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Heart to heart: Novo Nordisk teams with ACC to open diabetes discussion – FiercePharma

October 2nd, 2020 10:55 am

Novo Nordisk wants the heart disease community to join the diabetes conversation. As Type 2 diabetes drugs nab approvals for reduced cardiovascular risksincluding Novos own Ozempic and Victozathe need to communicate across specialties has grown.

To help bridge the gap, Novo Nordisk is partnering with the American College of Cardiology (ACC) in a two-year, two-pronged initiative. First, the partners will studyreal-world data to determine patterns and adherence to guidelines. Next, they'll explorehow to help Type 2 patients improve their heart health, workingthrough ACC clinicians and healthcare providers, Novo Chief Medical Officer Todd Hobbs said.

RELATED: Novo's Ozempic scores major win with heart-helping FDA approval. Is Rybelsus next?

Developing COVID-19 vaccines may not be enough: Turning vaccines into vaccinations

COVID-19 vaccines are being developed at a breakneck pace, but a broken supply chain could derail that momentum. What are the steps needed to help ensure the medical supply chain is up to the task?

The effort is a natural progression of Novo Nordisks relationships built up in the cardiovascular community over the past several years, he said.

Weve looked at smaller cuts of research, like with Cleveland Clinic and others, that show theres a lot of people out there with diabetes who really should be on one of the agents, either GLP-1 or SGLT2, that are proven to lower (cardiovascular) risk, and they just arent, Hobbs said.

While not an explicitpart of the study, COVID-19 has magnified the risks for people with diabetes and with heart disease. The partnership was in the works before the pandemic, but the novel coronavirus has made the initiative even more timely and relevant, Hobbs said.

The first part of the workevaluating data assessing adherence to ACC guidelines among a cohort of people with CV diseasewill be done within a year. The second stage will roll out specific treatment strategies to selected regional practices and hospitals, delivered through ACC healthcare ambassadors.

RELATED: Novo shifts Victoza ad message with spots highlighting heart-benefit trifecta

In January, Novo's next-gen GLP-1 drug Ozempic won an FDA toadd CV risk-reduction language to its label, specifying the benefits for patients with Type 2 diabetes andestablished cardiovascular disease.

Ozempic launched in early 2018 and took off quickly, nabbing $1.64 billion in 2019 sales. It's expected to reach $2.64 billion this year.

Novos newly launched Rybelsus, the oral version of Ozempic, is currently being studied as a CV preventive for patients with Type 2 diabetes. The company'solder GLP-1 diabetes med Victoza got a similar CV approval from the FDA in 2017 to add that it reduces the risk of heart attack, stroke and cardiovascular death.

While the ACC partnershipdoesnt includeany specific products, Hobbs said, If we can highlight the guidelines, whether thats ACC or ADA treatment guidelines, then our products will do well because theyre on the data.

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Diabetes raises the risk of liver damage in people with HIV – aidsmap

October 2nd, 2020 10:55 am

Almost half of people with HIV with unexplained liver enzyme elevations or other abnormal liver markers had NASH and a third had stage F3 or F4 fibrosis, and advanced liver damage was strongly associated with type 2 diabetes, a four-country study reports in Clinical Infectious Diseases.

Fatty liver disease occurs when fat builds up in the liver. Greater fat accumulation can lead to non-alcoholic steatosis (NASH), in which liver cells balloon and become inflamed. Unchecked, NASH leads to scarring of liver tissue (fibrosis) and eventually to cirrhosis, in which normal liver functions decline.

Although a high prevalence of non-alcoholic fatty liver disease (NAFLD) has been reported in people living with HIV, its unclear what proportion of these cases already have NASH or advanced fibrosis. The only way that these conditions can be diagnosed definitively is through liver biopsies (direct sampling of liver tissue).

Thickening and scarring of connective tissue. Often refers to fibrosis of the liver. See also cirrhosis, which is more severe scarring.

Non-alcoholic fatty liver disease (NAFLD) is a very common disorder and refers to a group of conditions where there is accumulation of excess fat in the liver of people who drink little or no alcohol. The most common form of NAFLD is a non-serious condition called fatty liver, by which fat accumulates in the liver cells. A small group of people with NAFLD may have a more serious condition named non-alcoholic steatohepatitis (NASH).

A procedure to remove a small sample of tissue so that it can be examined for signs of disease.

In NASH, fat accumulation is associated with liver cell inflammation and different degrees of scarring. NASH is a potentially serious condition that may lead to severe liver scarring and cirrhosis. It sometimes affects older people living with HIV.

A group of diseases characterized by high levels of blood sugar (glucose). Type 1 diabetes occurs when the body fails to produce insulin, which is a hormone that regulates blood sugar. Type 2 diabetes occurs when the body either does not produce enough insulin or does not use insulin normally (insulin resistance). Common symptoms of diabetes include frequent urination, unusual thirst and extreme hunger. Some antiretroviral drugs may increase the risk of type 2 diabetes.

To learn more about the frequency of NASH in people living with HIV, investigators at hospitals in the United Kingdom, Italy, Canada and the United States carried out a retrospective study of biopsy samples from 166 people with HIV who had been referred for investigation of unexplained increases in liver enzymes or other abnormalities in laboratory markers of liver disease between 2001 and 2019.

The analysis excluded people with hepatitis B or C, any active cancer, alcohol consumption above 21 units a week for men or 14 units for women, or any other cause of chronic liver disease.

Study participants had a median age of 48 years, 93% were male, 72% were white and the median duration of antiretroviral treatment was nine years. The median CD4 cell count was 638 cells/mm3. None had taken older nucleoside analogues associated with steatosis (stavudine, didanosine). The median body mass index was 29 kg/m2 (borderline obesity), 53% had high blood pressure and 25% had diabetes.

Biopsies showed that 63 of 116 people had NAFLD (54%) and 57 (49% of the entire cohort) had NASH. Thirty-six people (31%) had F3 stage fibrosis and three (2%) had F4 fibrosis (cirrhosis).

"Theinvestigators recommend consideration of liver biopsy as a screening tool in people with HIV who are obese, especially those with type 2 diabetes."

Multivariate analysis showed that after controlling for metabolic factors associated with NAFLD (model 1) or HIV-related factors (model 2), the only factor associated with NAFLD was higher body mass index (adjusted odds ratio 1.20 in both models, p=0.001).

Advanced fibrosis (F3 or above) was the only factor associated with type 2 diabetes in multivariate analysis (aOR 3.42, 95% CI 1.00-11.71) and this association was on the borderline of statistical significance (p=0.05).

Using biopsy results as a gold standard, investigators also assessed whether laboratory markers could accurately identify patients with advanced fibrosis. They found that both the FIB-4 and NAFLD Fibrosis scores performed poorly in identifying advanced fibrosis but showed good sensitivity (93%) in ruling out cases where the collagen proportionate area (CPA) was above 7.6%. CPA measures the percentage of liver tissue that is fibrotic and a level above 7.6% has been shown to predict long-term adverse liver disease outcomes.

The poor prognostic value of non-invasive markers for liver fibrosis in people living with HIV leads the investigators to recommend consideration of liver biopsy as a screening tool in people with HIV who are obese, especially those with type 2 diabetes.

However, they also note that 41% of people referred for biopsy did not have NAFLD. Their liver enzyme elevations remained unexplained but 15 of these 53 patients had advanced fibrosis (Ishak score 3 or above). The only factor associated with advanced fibrosis in those without NAFLD was time since HIV diagnosis (21 years vs 11.5 years, p= 0.005), even though age was similar between the two groups.

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Pulse Biosciences Announces FDA IDE Approval and Initiation of Sebaceous Hyperplasia Study – BioSpace

October 2nd, 2020 10:53 am

Oct. 1, 2020 11:30 UTC

Completes First Procedures in CellFX System Specific Indication Study

HAYWARD, Calif.--(BUSINESS WIRE)--Pulse Biosciences, Inc. (Nasdaq: PLSE), a novel bioelectric medicine company progressing Nano-Pulse Stimulation (NPS) technology, today announced FDA Investigational Device Exemption (IDE) approval and initiation of a pivotal study to evaluate the treatment of sebaceous hyperplasia (SH) lesions using the CellFX System. The data generated from this study is intended to support a 510(k) submission to expand the indication for use of the CellFX System specifically to treat SH lesions.

Following IDE approval, several patients have been enrolled, with the first patient procedure completed on September 28, 2020. The multicenter prospective comparative study is intended to evaluate the safety and efficacy of procedures to clear facial SH lesions performed with the CellFX System versus those performed by electrodessication in a comparator group. Enrollment of 60 patients across five study sites is expected to be completed in approximately three months. All subjects will have up to two treatments and will be evaluated through the primary safety and efficacy endpoints at 60-days following their last treatment. The ClinicalTrials.gov Identifier for the study is NCT04539886.

We are pleased to have received FDA IDE approval and to have begun this important SH comparative study slightly ahead of the fourth quarter start we had previously communicated. Understanding the COVID-19 pandemic has increased the demand on FDA resources, we appreciate their attention throughout the IDE process. Barring delays in enrollment, we expect to conclude the study in the first quarter of 2021 and plan to quickly follow with a 510(k) submission for the corresponding specific indication. We have long viewed SH as a top addressable market priority for the CellFX System based on patient demand in clinics today and the CellFX Systems early demonstration of procedure effectiveness, said Darrin Uecker, President and CEO of Pulse Biosciences. As we have communicated previously, in parallel we are completing our GLP preclinical study in support of the initial CellFX System 510(k) submission for a general dermatologic indication. We remain on track to submit this 510(k) in the next several weeks.

About Sebaceous Hyperplasia

Sebaceous hyperplasia (SH) is a very common skin condition that presents as shiny, yellowish or white raised bumps, or lesions, that most frequently occur on the face and are often oily in appearance. SH lesions form on the skin surface when the sebaceous glands, which are located in the deeper layer of the skin, become enlarged and form bumps between 2 and 4 millimeters wide on the facial skin surface. These deeper sebaceous glands that cause the SH lesion are difficult to treat with current thermal technologies without damaging the skin surface.

Based on a 2019 survey1, dermatologists who specialize in aesthetic procedures see an average of over 40 patients per week with SH lesions and their surveyed aesthetic patients diagnosed with this common problem are highly motivated to seek treatment as a cash-paying procedure to improve the appearance of their facial skin. Yet the majority of these patients diagnosed with SH are untreated, likely due to limitations of existing treatments that either cannot reach the sebaceous gland or that damage the skin surface, making the skin appearance worse than prior to treatment. Given the profile of NPS technology as a new option to reach these sebaceous glands with more desirable cosmetic effects, in the same survey, 88% of these aesthetic dermatology specialists reported a clear interest in a new procedure to address SH lesions. Previously published clinical data by the Company demonstrated that over 90% of SH lesions were cleared or mostly cleared by 60 days post-NPS treatment.

1 2019 Physician (n=304) and Patient (n=405) surveys conducted by third-party market research firm on behalf of Pulse Biosciences, Inc.

About Pulse Biosciences

Pulse Biosciences is a novel bioelectric medicine company committed to health innovation that has the potential to improve and extend the lives of patients. If cleared, the CellFX System will be the first commercial product to harness the distinctive advantages of the Companys proprietary Nano-Pulse Stimulation (NPS) technology to treat a variety of applications for which an optimal solution remains unfulfilled. Nano-Pulse Stimulation technology delivers nano-second pulses of electrical energy to non-thermally clear cells while sparing adjacent non-cellular tissue. Subject to regulatory approval, the initial commercial use of the CellFX System is expected to address a broad range of dermatologic conditions that share high demand among patients and practitioners for improved and durable aesthetic outcomes. Designed as a multi-application platform, the CellFX System is intended to offer customer value with a utilization-based revenue model across an expanding spectrum of clinical applications. To learn more please visit http://www.pulsebiosciences.com.

Pulse Biosciences, CellFX, Nano-Pulse Stimulation, NPS and the stylized logos are among the trademarks and/or registered trademarks of Pulse Biosciences, Inc. in the United States and other countries.

Caution: Pulse Biosciences CellFX System and Nano-Pulse Stimulation technology are for investigational use only.

Forward-Looking Statements

All statements in this press release that are not historical are forward-looking statements, including, among other things, statements relating to Pulse Biosciences expectations regarding regulatory clearance and the timing of FDA and other regulatory filings or approvals, including meetings with FDA and the ability of the Company to successfully complete a 510(k) submission for the CellFX System or for a specific indication for the treatment of sebaceous hyperplasia (SH) lesions, the ability of the Company to prepare and provide data to FDA and other regulatory bodies, NPS technology including the effectiveness of such technology and the effectiveness of related clinical studies in predicting outcomes resulting from the use of NPS technology, the CellFX System including the benefits of the CellFX System and commercialization of the CellFX System, current and planned future clinical studies and the ability of the Company to execute such studies and results of any such studies, other matters related to its pipeline of product candidates, the Companys market opportunity and commercialization plans, including the market for the treatment of SH, future financial performance, the impact of COVID-19 and other future events. These statements are not historical facts but rather are based on Pulse Biosciences current expectations, estimates, and projections regarding Pulse Biosciences business, operations and other similar or related factors. Words such as may, will, could, would, should, anticipate, predict, potential, continue, expects, intends, plans, projects, believes, estimates, and other similar or related expressions are used to identify these forward-looking statements, although not all forward-looking statements contain these words. You should not place undue reliance on forward-looking statements because they involve known and unknown risks, uncertainties, and assumptions that are difficult or impossible to predict and, in some cases, beyond Pulse Biosciences control. Actual results may differ materially from those in the forward-looking statements as a result of a number of factors, including those described in Pulse Biosciences filings with the Securities and Exchange Commission. Pulse Biosciences undertakes no obligation to revise or update information in this release to reflect events or circumstances in the future, even if new information becomes available.

View source version on businesswire.com: https://www.businesswire.com/news/home/20201001005320/en/

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Zebrafish Embryo Model Helps Understand the Workings of Nanoparticles in Blood – AZoNano

October 2nd, 2020 10:53 am

Written by AZoNanoOct 1 2020

When nanoparticles are injected into the bloodstream, for instance, to kill solid tumors, it is not known what exactly happens to them.

Now, with new results reported in the ACS Nano journal, scientists from Aarhus University are set to deal with this challenging query by using zebrafish embryos as a new research model in nanotoxicology and nanomedicine.

A wide range of nanoparticles are engineered for targeted drug delivery, but regrettably, just a few of the injected nanoparticles reach the site of target, such as solid tumors. The reason for this low targeting efficiency is frequently considered a black box and thus had not been investigated thoroughly for a number of years.

An international research group, headed by Yuya Hayashi from the Department of Molecular Biology and Genetics (MBG), Aarhus University, has now shown the beauty of zebrafish embryos in nano-bioimaging that can view dynamic interactions between nanoparticles and target cells in a living entity.

Currently, in association with scientists from Interdisciplinary Nanoscience Center (iNANO), Yuya aims to answer the unexplained mysteries in bionanosciencethe first one is the biological identity concept, which elucidates how cells detect nanoparticles via a corona of proteins surrounding each particle.

For the first time, this concept has now been demonstrated in a living organism through the use of zebrafish embryos, revealing what exactly occurs to nanoparticles when they are injected into the blood.

What the Cell Sees in Bionanoscience is one of the initial publications that have established how a protein corona develops around a nanoparticle and how this protein corona indicates the need for reconsidering the way one observes nanoparticles within a biological setting. From elaborate research made in the last several years, researchers have now understood that two opposing effects mostly play a role in the cellular uptake of nanoparticles.

Generally, the protein corona inhibits the surface of the nanoparticle from direct physical interactions with the cell membrane. But what if the protein corona sends a signal that activates a particular biological interaction with receptors positioned on the cell membrane? That is something the cell detects and therefore confers a biological identity to the nanoparticle.

Currently, the scientists from Aarhus University have thus given the first visual proof for the excellent influence of the protein corona with regard to the clearance of nanoparticles from the blood that involved adverse results in the zebrafish embryo model.

The researchers employed a species-mismatched source of proteins for the formation of corona to produce a non-self biological identity and traced the movement of nanoparticles traveling via the blood and to their final targetthat is, endolysosomes in the cell.

This showed an unexpectedly quick uptake and acidification of the nanoparticles by scavenger endothelial cells (functional counterpart to the liver sinusoidal endothelial cells in mammals) followed by pro-inflammatory stimulation of macrophages.

It sounds like a crazy idea to inject nanoparticles with proteins from another animal, but for example, biomolecule-inspired nanomedicines are tested in a mouse model without particular concerns for the species-mismatched combination.

Yuya Hayashi, Department of Molecular Biology and Genetics, Aarhus University

Hayashi continued, Or else some clever folks humanise the mouse to take care of the species compatibility problem. In fact, even at the cell culture level nanoparticles are still routinely tested following the tradition to use serum supplement derived from cows while knowing that nanoparticle-protein interactions are a key driver of cellular uptake.

What makes this kind of experiments rather challenging is to maximally retain the original protein corona in a living organism. If the pre-formed corona gets quickly exchanged by endogenous blood proteins, the hypothesis tested becomes invalid. We have made quite some efforts to characterise the protein corona to ensure the nanoparticles preserve the non-self-biological identity.

Hossein Mohammad-Beigi, Study First Author, Aarhus University

The maximum benefit of the zebrafish model is its power in multicolor immediate imaging, whereby numerous combinations of reporter proteins and fluorescence tracers can be viewed in a basic arrangement at high spatio-temporal resolution. This offers a new chance that lies between less lifelike cell culture systems and more complex rodent experiments, like intravital microscopy.

Using cell cultures, we have learnt quite a lot about how cells recognise nanoparticles rather as dynamic aggregates of proteins but it was never tested in a more realistic situation. With establishment of the zebrafish model, we have finally acquired a means to further explore this question in a living organism.

Yuya Hayashi, Department of Molecular Biology and Genetics, Aarhus University

It was a simple approach with an extreme scenario tested in a very complex system, but I believe we are now one step closer to understanding what the protein corona can really mean to nanoparticles. In an environment rich in proteins, nanoparticles can wear a mask that gives them a biological identity, and its non-selfness can make them a foe. What defines the degree of the non-selfness? Well, it's the next big question we have to address, Hayashi concluded.

Mohammad-Beigi, H., et al. (2020) Tracing the In Vivo Fate of Nanoparticles with a Non-Self Biological Identity. ACS Nano. doi.org/10.1021/acsnano.0c05178.

Source: https://mbg.au.dk/en

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Impact Of Covid-19 on Nanomedicine Market 2020 Industry Challenges, Business Overview and Forecast Research Study 2026 – Crypto Daily

October 2nd, 2020 10:53 am

Manhattan, New York, Analytical Research Cognizance: TheNanomedicineMarketreport is based on the basis of product type, application and end-user during the truncated forecast period. The detailed study further offers a broad interpretation on the Nanomedicine market based on a systematic analysis of the market from a variety of reliable sources and thorough data points. Furthermore, the report sheds a light on the Global scale segmenting the market space across various districts, appropriate distribution channels, generated income and a generalized market space.

This intelligence and 2025 forecasts Nanomedicine industry report further exhibits a pattern of analyzing previous data sources gathered from reliable sources and set a precedented growth trajectory for the Nanomedicine market. The report also focuses on a comprehensive market revenue streams along with growth patterns, analytics focused on market trends, and the overall volume of the market.

Request Sample of Global Nanomedicine Market Report @https://www.arcognizance.com/enquiry-sample/923566

Finally, the report provides detailed profile and data information analysis of leading Augmented Reality Company.

This report covers leading companies associated in Nanomedicine Market @GE Healthcare, Johnson & Johnson, Mallinckrodt plc, Merck & Co. Inc., Nanosphere Inc., Pfizer Inc., Sigma-Tau Pharmaceuticals Inc., Smith & Nephew PLC, Stryker Corp, Teva Pharmaceutical Industries Ltd., UCB (Union chimique belge) S.A

Region Segmentation:North America (U.S., Canada, Mexico)Europe (Germany, U.K., France, Italy, Russia, Spain etc.)Asia-Pacific (China, India, Japan, Southeast Asia etc.)South America (Brazil, Argentina etc.)Middle East & Africa (Saudi Arabia, South Africa etc.)

On the basis of types, the Nanomedicine market is primarily split into:Regenerative Medicine, In-vitro & In-vivo Diagnostics, Vaccines, Drug Delivery

On the basis of applications, the market covers:Clinical Cardiology, Urology, Genetics, Orthopedics, Ophthalmology

Some of the major factors contributing to the growth of the global Nanomedicine market:

Nanomedicine Market Report Structure at a Glance:

Access Global Nanomedicine Market Report @https://www.arcognizance.com/report/global-nanomedicine-market-status-and-future-forecast-2015-2025

Table of Content:

Note:Our report does take into account the impact of corona virus pandemic and dedicates qualitative as well as quantitative sections of information within the report that emphasizes the impact of COVID-19.

As this pandemic is ongoing and leading to dynamic shifts in stocks and businesses worldwide, we take into account the current condition and forecast the market data taking into consideration the micro and macroeconomic factors that will be affected by the pandemic.

About us:Analytical Research Cognizance (ARC) is a trusted hub for research reports that critically renders accurate and statistical data for your business growth. Our extensive database of examined market reports places us amongst the best industry report firms. Our professionally equipped team further strengthens ARCs potential. ARC works with the mission of creating a platform where marketers can have access to informative, latest and well researched reports. To achieve this aim our experts tactically scrutinize every report that comes under their eye.

Contact Us:Ranjeet DengaleDirector SalesAnalytical Research Cognizance+1 (646) 403-4695, +91 90967 44448[emailprotected]

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Cristal Therapeutics announces a publication in ‘Chemical Science’ on CliCr technology platform, comprising a new class of superior copper free click…

October 2nd, 2020 10:53 am

For the optimal performance of CriPec nanomedicines, it is essential to be able to attach a broad range of small molecule active agents and large molecular entities, biologics, to CriPec nanoparticles.

The published research1reports the development of a convenient and versatile fast-reacting molecular entity for gluing very different compounds in a strain-promoted azide-alkyne cycloaddition click reaction to the nanoparticles, as well as a collection of linkers to attach the widely varying active small molecules and biologics. Next to the already demonstrated examples, many additional applications are foreseen such as the construction of antibody drug conjugates in aqueous environments with faster kinetics that is essential for these delicateconstructs.

CliCr is also used to generate virus mimicking nanoparticles. CriVac is a unique antigen carrier platform based on CriPec nanoparticles that, in contrast to viral vectors, do not convey a bystander immune response. CriPec particles' size resemble a virus and the desired numbers of antigen displayed on its surface are controlled via CliCr. CriVac mimics features of a live virus in a tailored manner to induce immunity safely, efficiently and solely to the displayed antigen, offering a prophylactic vaccination strategy which will be readily adaptable to different pathogenic treats.

The very attractive functionalisation possibilities, combined with its versatility, great reactivity and small size offer multiple opportunities for CliCrreagents to become the new standard for non-copper catalyzed click reactions in a multitude of applications.

Dr Cristianne Rijcken, CSO of Cristal Therapeutics, stated:

"This new versatile click reagent originates from an intense collaboration between industry and academic partners. For our nanomedicine applications, a fast, cleanly reacting and small click reagent is absolutely indispensable. These demands required the development of a new reagent, which will be highly attractive both for our proprietary applications and for the wider world of the biological, medical and material science applications. This is ground-breaking technology!"

In case you are interested to learn about the CliCr platform, please reach out to http://www.clicr.euor talk to us at the following virtual conferences

Reference

1. J. Weterings et al. TMTHSI, a superior 7-membered ring alkyne containing reagent for strain-promoted azidealkyne cycloaddition reactions, Chemical Science (2020)

https://pubs.rsc.org/en/content/articlehtml/2020/sc/d0sc03477k

About Cristal Therapeutics

Cristal Therapeutics is a phase 2 clinical-stage pharmaceutical company developing targeted nanomedicines for the treatment of cancer and other diseases with high unmet patient need and considerable commercial potential. The Company's product candidates are based on its proprietary CriPec polymeric nanoparticle technology platform, which enables the design of customized nanomedicines with superior therapeutic profiles. CriPec-based products have the potential to provide enhanced efficacy and reduced side effect profiles, thus offering improved disease treatment.

Find out more: http://www.cristaltherapeutics.com

For more information, please contact:

Cristal Therapeutics Jeroen van EgmondConsultant Business DevelopmentT: +31 6 272 048 89E: [emailprotected]

SOURCE Cristal Therapeutics

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UH Manoa scholars awarded ARCS Foundation grants for research – UH System Current News

October 2nd, 2020 10:53 am

Top for from left: Douglas Ellman, Branden Minei, Luke Campillo, Allexa Dow; Middle row from left: Marisa McDonald, Ashley McGuigan, John Runburg, Michael Honda, Priscilla Seabourn, Lauren Ching; Bottom row from left: Aileen Li, Brien Haun, Anamica Bedi de Silva, Trista McKenzie, Gagandeep Anand, Travis Berger

Sixteen University of Hawaii at Mnoa doctoral candidates have been awarded $5,000 Scholar Awards from the ARCS FoundationHonolulu Chapter. The 2020 awards were made in six UH Mnoa units.

ARCS Foundation works to advance science in America by providing unrestricted awards to outstanding U.S. graduate students in STEM fields. The chapter has provided more than $2 million to UH graduate students since 1974.

This award has not only provided monetary support for my research, but it shows that theres recognition for my work outside of my immediate sphere, and thats very meaningful, says ARCS Scholar Trista A. McKenzie.

McKenzie and her award donor are featured in the Honolulu Chapters first Meet-the-Scholar video, which was created after COVID-19 preempted the annual scholar presentations.

Douglas Ellman received the Bretzlaff Foundation Award in Engineering. He uses optimization and machine learning to study how distributed energy resources, such as solar batteries, electric vehicles and smart appliances, can be used to improve the operation of the electric grid.

Brenden M. Minei received the Frederick M. Kresser Award in Engineering. He developed a novel ceramic-based Nano-Paste that can be both 3D printed and molded to optimize and develop ceramic nanocomposite parts with armor as well as space structure and optical applications.

Read more about the College of Engineering scholars.

Luke Campillo received the Sarah Ann Martin Award in Natural Sciences. He sequences the DNA Hawaiian birds to study the impact of limited contact with other populations and competition for limited resources on speciation on island archipelagos.

Allexa Dow received the Ellen M. Koenig ARCS Award. She studies mechanisms employed by the deadly Mycobacterium tuberculosis pathogen to survive severe zinc depletion in the host, a necessary step for disease transmission.

Marisa S. McDonald received the Maybelle Roth ARCS Award in Conservation Biology. She is working to understand vision in larval mantis shrimp, focusing on ultraviolet vision function and use.

Ashley A. McGuigan received the ARCS Honolulu Award. She explores the connection between agroforest biodiversity and dietary nutrition in Fiji and the ways agroforestry helps people recover from major cyclone disturbances.

John Jack Runburg received the Sarah Ann Martin Award in Natural Sciences. He uses theoretical models and devises other methods to learn more about dark matter, the most common, but invisible, form of matter in the universe.

Read more about the College of Natural Sciences scholars.

Michael David Honda received the Kai Bowden ARCS Award. He is working to determine the mechanism of iron uptake in giant leucaena, which is used as a nutritious fodder-legume for farm animals.

Priscilla S. Seabourn received the Helen Jones Farrar Award. She uses DNA sequencing to characterize the microbiome and understand how environmental and ecological factors influence its diversity in Aedes albopictus mosquitoes, an insect that serves as a vector for Dengue and Chikungunya viruses, with an eye to improving strategies for disease prevention.

Read more about the CTAHR scholars.

Lauren Ching received the Koenig ARCS Award. She studies immunopathogenesis of Kawasaki disease, the leading cause of pediatric acquired heart disease in the developed world, to identify novel therapeutics that could ameliorate changes to coronary arteries.

Aileen S.W. Li received the Starbuck ARCS Award in Medicine. She uses in vitro model systems to understand the mechanisms of gastrulation, the foremost, crucial and sensitive stage of embryo development, and exposure to teratogens, agents that can cause birth defects.

Brien Haun received the Ellen M. Koenig ARCS Award. He is working to hack the immune system to uncover protective responses to emerging infectious viruses.

Read more about the JABSOM scholars.

Anamica Bedi de Silva received the George and Mona Elmore ARCS Award. She works on viral immunity in microbes, developing resistant strains of a single-cell algae for experimental evolution in the laboratory to determine if there are fitness costs to viral resistance.

Trista A. McKenzie received the Toby Lee ARCS Award in Earth Sciences. She studies groundwater pollution and discharge dynamics using a combination of field, lab and machine-learning approaches.

Read more about the SOEST scholars.

Gagandeep Deep Anand received the ARCS Honolulu Award. He is determining accurate distances to nearby galaxies using Hubble Space Telescope imaging to investigate the distribution of matter and evolution of galaxy groups and clusters.

Travis A. Berger received the Columbia Communications Award in Astronomy. He studies planet formation and evolution using stellar distances as measured by the Gaia space observatory of stars and exoplanets observed by the Kepler space telescope.

Read more about the IfA scholars.

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The Europe exosome diagnostic and therapeutic market is projected to reach US$ 12,524.24 thousand in 2019 to US$ 104,694.72 thousand by 2027 -…

October 2nd, 2020 10:53 am

New York, Oct. 01, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Europe Exosome Diagnostic and Therapeutic Market Forecast to 2027 - COVID-19 Impact and Regional Analysis By Application ; Product ; End User, and Country" - https://www.reportlinker.com/p05974359/?utm_source=GNW

Exosome is an emerging industry with a huge potential.Applications of exosomes are expanding rapidly in the areas of disease diagnosis and treatment as well as pharmaceuticals.

Exosomes are nanovesicles and act as a vehicle to deliver therapies to cells of the body.In the future, exosomes can be used as potential biomarkers and in the field of personalized medicine.

Interest in exosome research has increased dramatically in recent years, driving the growth of the exosome diagnostic and therapeutic market in the UK.New exosome therapeutics companies are rapidly entering the marketplace.

The investment flow has also increased to support such innovative therapeutic companies, further boosting the growth of the market. For example, in 2016, ReNeuron Group plc, a leading UK-based stem cell therapy development company, was awarded about US$ 2.6 million grant from Innovate UK to advance its emerging exosome nanomedicine platform.In terms of application, the diagnostics application segment accounted for a larger share of the Europe exosome diagnostic and therapeutic market. Its growth is attributed to an increasing adoption of exosome-based instruments and kits for diagnosis of chronic conditions. Additionally, exosome-based diagnostic products offer benefits such as accuracy, lower processing time, and better ergonomics; these are likely to drive the growth of diagnostic application segment in the Europe exosome diagnostic and therapeutic marketIn 2019, the instrument segment held a considerable share of the for exosome diagnostic and therapeutic market, by the product.This segment is also predicted to dominate the market by 2027 owing to higher demand for diagnostics instruments.

However, the software segment is anticipated to witness growth at a significant rate during the forecast period.A few major primary and secondary sources for the exosome diagnostic and therapeutic market included in the report are Instrument, US Food and Drug Administration, and World Health Organization, among others.Read the full report: https://www.reportlinker.com/p05974359/?utm_source=GNW

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The Europe exosome diagnostic and therapeutic market is projected to reach US$ 12,524.24 thousand in 2019 to US$ 104,694.72 thousand by 2027 -...

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Nanorobotics Market to Witness Huge Growth by 2024 | Bruker, JEOL, Thermo Fisher Scientific, Ginkgo Bioworks, Oxford Instruments – The Daily Chronicle

October 2nd, 2020 10:53 am

Global Nanorobotics Market report study covers the breakdown data with production, consumption, revenue and market share by regions, type and applications. Historical breakdown data from 2015 to 2019 and forecast to 2024

The comprehensive numerical analyses of Global NanoroboticsMarket Research Report 20202024 is a historical overview and in-depth study on the current & future market of the Nanorobotics industry. he report focuses on the historical and current market trends to predict the course of the global Nanorobotics market in the upcoming years. The report identifies opportunities, drivers, and major challenges faced by market players. The report discusses all major market aspects with expert opinion on current market status along with historic data. This market report is a detailed study on the growth, investment opportunities, market statistics, growing competition analysis, major key players, industry facts, important figures, sales, prices, revenues, gross margins, market shares, business strategies, top regions, demand, and developments. The research further provides par excellence futuristic estimations for various vital factors including market size, share, net profit, sales, revenue, and growth rate. The market competition by top manufacturers/players, with sales volume, price, revenue (Million USD) and market share for each manufacturer/player; the top players including market:Bruker, JEOL, Thermo Fisher Scientific, Ginkgo Bioworks, Oxford Instruments, EV Group, Imina Technologies, Toronto Nano Instrumentation, Klocke Nanotechnik, Kleindiek Nanotechnik. This report provides in-depth analysis of the Nanorobotics market and provides market size (US$ million) and compound annual growth rate (CAGR %) for the forecast period (20202024).

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Global major manufacturers of the market are also assessed with their information such as company profiles, product picture and specification, capacity, production, price, cost, market trend, revenue, and contact data. The research provides details regarding each product like the cost breakup, import/export scheme, manufacturing volume, price, gross, growth ratio, investments, and contribution to the global Nanorobotics revenue. The facts and data are represented in the Nanorobotics Market report using diagrams, graphs, pie charts, and other pictorial representations. This enhances the visual representation and also helps in understanding the facts much better. We have provided a detailed study on the critical dynamics of the global Nanorobotics market, which include the market influence and market effect factors, drivers, challenges, restraints, trends, and prospects. Global Nanorobotics Industry Market Research Report is providing exclusive vital statistics, information, data, trends and competitive landscape details. The research study also includes other types of analysis such as qualitative and quantitative. The document also comprises of a detailed assessment of the regional scope of the market alongside its regulatory outlook. Additionally, the report provides with a detailed SWOT analysis while elaborating market driving factors. Furthermore, it sheds light on the comprehensive competitive landscape of the global market. Nanorobotics market report further offers a dashboard overview of leading companies encompassing their successful marketing strategies, market contribution, recent developments in both historic and present contexts.

The Nanorobotics market report includes the overall and comprehensive study of the Nanorobotics market with all its aspects influencing the growth of the market. This report is exhaustive quantitative analyses of the Nanorobotics industry and provides data for making strategies to increase the market growth and effectiveness. The Market report lists the most important competitors and provides the insights strategic industry Analysis of the key factors influencing the market. This report will help you to establish a landscape of industrial development and characteristics of the Nanorobotics market. The Global Nanorobotics market analysis is provided for the international markets including development trends, competitive landscape analysis, and key regions development status. It also provides statistical data on all the recent developments in the market. It also comprises a basic overview and revenue and strategic analysis under the company profile section. Nanorobotics market analysis is provided for the international markets including development trends, competitive landscape analysis, investment plan, business strategy, opportunity, and key regions development status. 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.

Regional Analysis:This section of the report contains detailed information on the market in different regions. Each region offers a different market size because each state has different government policies and other factors. The regions included in the report areNorth America (United States, Canada and Mexico), Europe (Germany, France, UK, Russia and Italy), Asia-Pacific (China, Japan, Korea, India, Southeast Asia and Australia), South America (Brazil, Argentina, Colombia), Middle East and Africa (Saudi Arabia, UAE, Egypt, Nigeria and SouthAfrica)Information about the different regions helps the reader to better understand the global Nanorobotics market.

Most important types of the market covered in this report are:Nanomanipulator, Bio-Nanorobotics, Magnetically Guided, Bacteria-Based

Most widely used downstream fields of market covered in this report are:Nanomedicine, Biomedical, Mechanical

Research objectives: The points that are discussed within the Nanorobotics Market report are the major market players that are involved in the market such as manufacturers, raw material suppliers, equipment suppliers, end users, traders, distributors and etc. Data and information by manufacturer, by region, by type, by application and etc, and custom research can be added according to specific requirements. The complete profile of the companies is mentioned. And the capacity, production, price, revenue, cost, gross, gross margin, sales volume, sales revenue, consumption, growth rate, import, export, supply, future strategies, and the technological developments that they are making are also included within the report. To analyze the Nanorobotics with respect to individual growth trends, future prospects, and their contribution to the total market. Focuses on the key global Nanorobotics manufacturers, to define, describe and analyze the sales volume, value, market share, market competition landscape, SWOT analysis and development plans in next few years. To project the consumption of Nanorobotics submarkets, with respect to key regions (along with their respective key countries). To strategically profile the key players and comprehensively analyze their growth strategies. The growth factors of the market are discussed in detail wherein the different end users of the market are explained in detail. The Nanorobotics market report contains the SWOT analysis of the market. Finally, the report contains the conclusion part where the opinions of the industrial experts are included.

Key Questions Answered: What is the size and CAGR of the global World Nanorobotics Market? Which are the leading segments of the global World Nanorobotics Market? What are the key driving factors of the most profitable regional market? What is the nature of competition in the global World Nanorobotics Market? How will the global Home Appliance market advance in the coming years? What are the main strategies adopted in the global World Nanorobotics Market? What are sales, revenue, and price analysis by types and applications of Nanorobotics market? What are sales, revenue, and price analysis by regions of Nanorobotics industry?

The Essential Content Covered in the Global Nanorobotics Market Report :* Top Key Company Profiles.* Main Business and Rival Information* SWOT Analysis and PESTEL Analysis* Production, Sales, Revenue, Price and Gross Margin* Market Share and Size

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The Nanorobotics market report enumerates information about the industry in terms of market share, market size, revenue forecasts, and regional outlook. The report further illustrates competitive insights of key players in the business vertical followed by an overview of their diverse portfolios and growth strategies. This report is comprehensive numerical analyses of the Nanorobotics industry and provides data for making strategies to increase the market growth and success. The Report also estimates the market size, Price, Revenue, Gross Margin and Market Share, cost structure and growth rate for decision making. A detailed evaluation of the market by highlighting information on different aspects which include drivers, restraints, opportunities, threats, and global markets including progress trends, competitive landscape analysis, and key regions expansion status.

At last, This report investigates the Nanorobotics market in the global market, presents the latest business analysis including market scope, product situation, technology growth, environmental distribution, business situation, and chain structure. industrial. Nanorobotics Market Report Shares Important Data on Impact Factors, Advertising Drivers, Challenges, the report gives the inside and out examination of Nanorobotics Market took after by above components, which are useful for organizations or individual for development of their present business or the individuals who are hoping to enter in Nanorobotics industry.

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Nanorobotics Market to Witness Huge Growth by 2024 | Bruker, JEOL, Thermo Fisher Scientific, Ginkgo Bioworks, Oxford Instruments - The Daily Chronicle

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Thalassemia Treatment Market projected to expand at a CAGR of 7.9% from 2018 to 2026 – The Daily Chronicle

September 30th, 2020 4:53 pm

Transparency Market Research (TMR)has published a new report titled, Thalassemia Treatment Market Global Industry Analysis, Size, Share, Growth, Trends, and Forecast, 20182026.According to the report, theglobal thalassemia treatment marketwas valued at US$ 842.0 Mn in 2017 and is projected to expand at a CAGR of 7.9% from 2018 to 2026. Increase in R&D investment by key players for developing new drugs for treating thalassemia and rise in demand for chelating therapy are anticipated to fuel the growth of the global market from 2018 to 2026. Asia Pacific and Middle East & Africa are expected to dominate the global market owing to increase in prevalence of thalassemia disorder and high adoption of chelation therapy & blood transfusion for treatment by doctors as well as patients. The market in Asia Pacific is projected to expand at the fastest CAGR during the forecast period. Growth of the market in the region is attributed to large base of private clinics and hospitals, rise in number of thalassemia population requiring chelation therapy services after spleen surgery, and surge in adoption of blood transfusion among patients. The thalassemia treatment market in Latin America is likely to expand at a moderate growth rate during the forecast period.

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Value Added Features in Thalassemia Treatments to Propel Global Market

The global thalassemia treatment market is projected to be driven by value added features offered by various thalassemia drug manufacturing companies in order to streamline the day to day work flow and increase revenue. The thalassemia treatment provides limited range of features and benefits ranging from patient pain heeling remedies to treatment procedures. For instance, very less number of people go for the much beneficial chelation therapy. These features help physicians and nurses to streamline the chelation therapy required for patients to maintain their daily workflow efficiently and effectively. Key players offering thalassemia treatment are coming up with value added features such as bone marrow transplantation, stem cell regeneration, gene editing methodologies, and effective modality features used for drug manufacturing along with creating a prominent candidate molecule for drugs. These features can reduce the overall operating cost and improve the overall effectiveness and efficiency of treatment practices. Companies are focusing on the development of combined drug therapy in their system to effectively integrate chelating therapy or other treatment procedure at an affordable cost. These value added features save time for physicians and help improve thalassemia patient survival performance.

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Chelation Therapy to be Highly Lucrative Segment

Traditionally, blood transfusion based on type of thalassemia treatment was the most commonly used procedure among thalassemia patients. This treatment type was associated with availability of donor and cost of treatment procedure. Moreover, chelation therapy based on thalassemia treatment are priced on perpetual license model and are expensive. Chelation therapy treatment enables patients to practice intensive therapy to treat acute iron overload leading to 90% recovery among thalassemia patients. These chelation therapy based treatments address specific challenges faced during the treatment procedure. The chelation therapy treatment facilitates benefits such as pain relief, and increase in motion of blood flow among patients.

Asia Pacific Presents Significant Opportunities

North America and Europe accounted for major share of the global thalassemia treatment market in 2017 and are likely to gain market shares by 2026. High rate of immigration from tropical regions, increasing health care budgets by governments, and government initiatives to promote thalassemia treatment technique contributed to the leading share of these regions. Asia Pacific is projected to be the most attractive market for thalassemia treatment, with highest attractiveness index. The market in Asia Pacific is expected to expand at a high CAGR of 9% during the forecast period due to large number of thalassemia patients opting for chelation therapy in developing countries such as India and China. Well-established health care facilities, medical tourism for treatment of thalassemia, and high adoption of blood transfusion safety technique in countries such as Turkey and GCC Countries are likely to drive the market in Middle East & Africa. The market in Latin America is poised to expand at a moderate growth rate during the forecast period.

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Trend of R&D among Leading Players to Increase Geographic Presence

The report also provides profiles of leading players operating in the global thalassemia treatment market. bluebird bio, Inc., Acceleron Pharma, Inc., Novartis AG, Celgene Corporation, and Shire plc (Takeda Pharmaceuticals) are the leading players operating in the global market. Companies operating in the thalassemia treatment market aim to increase geographic presence and research & development through strategic acquisitions and collaborations with leading players in respective domains and region. In December 2017, Shire plc committed to pay approximately US$ 1,409.9 Mn to contract vendors for administering and executing clinical trials. Other prominent players operating in the global thalassemia treatment include Incyte Corporation, Kiadis Pharma, Gamida Cell, Celgene Corporation, and Bellicum Pharmaceuticals.

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Thalassemia Treatment Market projected to expand at a CAGR of 7.9% from 2018 to 2026 - The Daily Chronicle

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Mild to severe: Immune system holds clues to virus reaction – ABC News

September 30th, 2020 4:51 pm

One of COVID-19's scariest mysteries is why some people are mildly ill or have no symptoms and others rapidly die and scientists are starting to unravel why.

An international team of researchers found that in some people with severe COVID-19, the body goes rogue and attacks one of its own key immune defenses instead of fighting the coronavirus. Most were men, helping to explain why the virus is hitting men harder than women.

And separate research suggests that children fare better than adults thanks to robust first responder immune cells that wane with age.

They're the latest in a list of studies uncovering multiple features of the immune system's intricate cascade that can tip the scales between a good or bad outcome. Next up: Figuring out if all these new clues might offer much-needed ways to intervene.

We have the knowledge and capability of really boosting many aspects of the immune system. But we need to not use the sledge hammer, cautioned Dr. Betsy Herold of New York's Albert Einstein College of Medicine, who co-authored the child study.

Adding to the complexity, people's wildly varying reactions also reflect other factors, such as how healthy they were to begin with and how much of the virus the dose" they were exposed to.

Infection and what happens after infection is a very dynamic thing, said Alessandro Sette, a researcher at the La Jolla Institute for Immunology in San Diego, who is studying yet another piece of the immune response.

IMMUNE PRIMER

There are two main arms of the immune system. Innate immunity is the bodys first line of defense. As soon as the body detects a foreign intruder, key molecules, such as interferons and inflammation-causing cytokines, launch a wide-ranging attack.

Innate immune cells also alert the slower-acting adaptive arm of the immune system, the germ-specific sharpshooters, to gear up. B cells start producing virus-fighting antibodies, the proteins getting so much attention in the vaccine hunt.

But antibodies aren't the whole story. Adaptive immunity's many other ingredients include killer T cells that destroy virus-infected cells and memory T and B cells that remember an infection so they spring into action quicker if they encounter that germ again.

A MISSING PIECE

Usually when a virus invades a cell, proteins called Type I interferons spring into action, defending the cell by interfering with viral growth. But new research shows those crucial molecules were essentially absent in a subset of people with severe COVID-19.

An international project uncovered two reasons. In blood from nearly 1,000 severe COVID-19 patients, researchers found 1 in 10 had what are called auto-antibodies antibodies that mistakenly attack those needed virus fighters. Especially surprising, autoimmune disorders tend to be more common in women but 95% of these COVID-19 patients were men.

The researchers didn't find the damaging molecules in patients with mild or asymptomatic COVID-19.

In another 660 severely ill patients, the same team found 3.5% had gene mutations that didn't produce Type I interferons.

Each of those silent vulnerabilities was enough to tip the balance in favor of the virus early on, said Dr. Jean-Laurent Casanova, an infectious disease geneticist at Rockefeller University in New York, who co-leads the COVID Human Genetic Effort.

Certain interferons are used as medicines and are under study as a possible COVID-19 treatment; the auto-antibody discovery adds another factor to consider.

KIDS' IMMUNITY REVS FAST

It's not clear why children appear less at risk from COVID-19. But occasionally they're sick enough for hospitalization, giving Herold's team the opportunity to compare 60 adults and 65 children and teens at New Yorks Montefiore Health System.

The children produced much higher levels of certain cytokines that are among the innate immune system's first responders. When the immune system's next stage kicked in, both adults and children made antibodies targeting the coronavirus. Here's the rub: The adults' adaptive immune response was more the type that can trigger an inflammatory overreaction.

The findings suggest kids' early robust reaction lets their immune system get ahead of the virus, making an overreaction less likely "and that's protecting them, Herold said.

ANY PREEXISTING IMMUNITY?

The coronavirus that causes COVID-19 is new to humans. But Sette's team studied blood samples that were stored in freezers before the pandemic and found some harbored memory T cells that recognized a tiny portion of the new virus in laboratory tests.

You can actually tell that this is an experienced T cell. This has seen combat before, Sette said. Researchers in Germany, Britain and other countries have made similar findings.

The new coronavirus has cousins that cause as many as 30% of common colds, so researchers believe those T cells could be remnants from past colds.

But despite the speculation, we don't know yet that having those T cells makes any difference in who gets seriously sick with COVID-19, noted Rory de Vries, co-author of a study in the Netherlands that also found such T cells in old blood.

All these findings beg for a deeper understanding of the myriad ways some people can be more susceptible than others.

We need to look quite broadly and not jump into premature conclusions about any one particular facet of the immune system, said Stanford University immunologist Bali Pulendran. He also has found some innate immune cells in a state of hibernation in seriously ill adults and next is looking for differences before and after people get sick.

But, it's not just all about the immune system, cautioned Dr. Anita McElroy, a viral immunity expert at the University of Pittsburgh Medical Center whos closely watching the research. A way to tell in advance who's most at risk? Were a long, long way from that.

The Associated Press Health and Science Department receives support from the Howard Hughes Medical Institutes Department of Science Education. The AP is solely responsible for all content.

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The immune system: How to boost it and lower your immune age – New Scientist News

September 30th, 2020 4:51 pm

Your immune system stands between you and deadly infections. But as you get older so does your immune age, making you more susceptible to disease. Fortunately, we are discovering plenty of things you can do to turn back the clock and stay healthy. In this episode of our video series Science with Sam, find out how your immune system works and how you can give it a boost.

Tune in every week toyoutube.com/newscientistfor a new episode, or check back tonewscientist.com

One of the most important things standing between you and a deadly infection is your immune system the intricate, biological defence mechanism that protects your body from harmful invaders. And theres a lot we can do to give our immune system a helping hand.

Your immune system is made up of two divisions: the innate immune system and the adaptive immune system, each with its own battalion of specialist cells and defensive weapons.

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The innate immune system is the first line of defence. Its made up of cells like the scary-sounding macrophage, and the less scary-sounding neutrophil. These general-purpose guards patrol the bloodstream on the lookout for anything that shouldnt be there. When they detect an intruder, they neutralise the threat by engulfing it like Pac-Man, spraying it with deadly chemicals or suicidally expelling their DNA and throwing it around the invader like a net.

Then theres the adaptive immune system, which you can think of as the immune systems special forces, elite agents trained to fight specific pathogens. Unlike the innate system, which can attack any invading cell or virus, these cells are only effective against one enemy, and they must be trained to fight them first.

B cells fight bacteria and viruses by making Y-shaped proteins called antibodies that neutralise an invader or tag it for attack by other parts of the immune system.

Then there are T cells. These coordinate and carry out attacks on infected cells. Helper T Cells call in reinforcements by sending out chemical messages known as cytokines. Killer T-Cells are the front line soldiers, trained, as the name suggests, to destroy the enemy.

When we encounter a disease for the first time, it takes a while for the adaptive immune system to learn how to fight it. But once its up and running, it creates a memory, allowing a fast and brutal response to future infections often neutralising it before you even notice. This is the premise of vaccines and the reason why you only get diseases like chicken pox once.

If you want to know more about vaccines, theres a video all about them, just hit the link at the end of this video. Better yet, subscribe to New Scientist today and get 20 per cent off if you enter the code SAM20 at checkout.

Your immune system works so well that, most of the time, you wont even notice it. But it weakens as you get older, making you more susceptible to infection. Thats a key reason why people over the age of 70 are most vulnerable to diseases like covid-19, or even the flu.

This decline happens to all of us, but it can be accelerated by lifestyle factors like smoking and inactivity. Obesity is also linked to a faster decline in immune potency.

All of which means that, although the strength of your immune system is linked to your age, a 40-year-old can have the immune system of a 60-year-old. Or on the flipside, a healthy 60-year-old may have the immune system of a 40-year-old.

Scientists have recently developed ways to measure your immune age. Fortunately, it turns out your immune age can go down as well as up. And there are some simple ways to turn back the clock on your immune system.

As we get older, some of our immune cells start to misbehave. Take neutrophils, those early responder cells. As they age, they get worse at hunting down intruders, blundering through your tissues, causing damage.

The root of the problem is an overactive enzyme involved in their sense of direction. Dialling down that enzyme rejuvenates the neutrophils so they know where theyre going. And theres a simple, drug-free way to do it: exercise.

One study in older adults showed that those who got 10,000 steps a day on average had neutrophils as good as a young adult.

Exercise also has benefits for your T cells. Before they are released onto active duty, T-cells mature in a little-known organ called the thymus gland in your chest. The thymus degenerates over time, resulting in a drop-off in the number of T cells.

Physical activity has a huge effect on the rate of this degeneration. A study found that amateur cyclists aged between 55 and 79 had youthful thymus glands and their T-cell counts were similar to those of much younger people.

Another key factor in your immune age is your gut bacteria. There is good evidence that poor gut health is a cause of premature ageing and that a healthy microbiome can reduce your immune age. Eating a healthy, varied diet rich in fibre, plant matter and fermented foods can help maintain a healthy community of gut microbes.

Your body has a highly evolved, intricate defence system thats effective at keeping you well, but only if you look after it.

I dont know about you but Ive been a bit less active of late, so Im considering this something of a wake-up call.

Looking after your immune system is a no-brainer, and its as easy as a walk in the park.

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Strong activation of anti-bacterial cells of immune system linked to severe Covid-19: Study – Hindustan Times

September 30th, 2020 4:51 pm

A type of the immune systems T cells known to fight against bacterial infections is strongly activated in people with moderate to severe Covid-19, according to a study which provides a better understanding of how the body responds to the novel coronavirus infection.

Researchers, including those from the Karolinska Institutet in Sweden, noted that this component of the immune system called MAIT cells make up about one to five percent of T cells in the blood of healthy people, and are primarily important for controlling bacteria, but can also be recruited to fight some viral infections.

They explained that T cells are a type of white blood cells that are specialised in recognizing infected cells, and are an essential part of the immune system. In the current study, published in the journal Science Immunology, the scientists assessed the role played by MAIT cells in Covid-19 disease.

They examined the presence and character of MAIT cells in blood samples from 24 patients admitted to Karolinska University Hospital with moderate to severe Covid-19 disease, and compared these with blood samples from 14 healthy controls and 45 individuals who had recovered from Covid-19. Four of the patients died in the hospital, the study noted.

To find potential treatments against Covid-19, it is important to understand in detail how our immune system reacts, and in some cases, perhaps contribute to worsening the disease, said Johan Sandberg, a co-author of the study at Karolinska Institutet.

According to the study, the number of MAIT cells in the blood decline sharply in patients with moderate or severe Covid-19, and the remaining cells in circulation are highly activated.

Based on these results, the scientists suggested that the MAIT cells are engaged in the immune response against the novel coronavirus SARS-CoV-2. This pattern of reduced number and activation in the blood is stronger for MAIT cells than for other T cells, they said. The study also noted that pro-inflammatory MAIT cells accumulated in the airways of Covid-19 patients to a larger degree than in healthy people.

Taken together, these analyses indicate that the reduced number of MAIT cells in the blood of Covid-19 patients is at least partly due increased accumulation in the airways, Sandberg said.

The scientists added that the number of MAIT cells in the blood of convalescent Covid-19 patients recovered at least partially in the weeks after disease, which can be important for managing bacterial infections in individuals who have had Covid-19. They said the MAIT cells tended to be extremely activated in the patients who died.

The findings of our study show that the MAIT cells are highly engaged in the immunological response against Covid-19, Sandberg said. The scientists believe the characteristics of MAIT cells make them engaged early on in both the systemic immune response, and in the local immune response in the airways to which they are recruited from the blood by inflammatory signals.

There, they are likely to contribute to the fast, innate immune response against the virus. In some people with Covid-19, the activation of MAIT cells becomes excessive and this correlates with severe disease, Sandberg added.

(This story has been published from a wire agency feed without modifications to the text.)

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ETHZ-led group shows that nervous system directly influences immune system – Optics.org

September 30th, 2020 4:51 pm

29Sep2020

Optogenetics researchers in Switzerland and US have optically stimulated nerve fibers in living mice.

Through this process, they have demonstrated that the nervous system has a direct influence on the immune system.

Over the past ten years, a new method has literally shed more light on the brain: optogenetics allows scientists to dstimulate genetically-modified nerve cells and investigate their functioning within the complex network inside the skull. This technique represents a revolution in neuroscience, say the Swiss-US team, but to date, it could only be applied to study the central nervous system, and not the peripheral nervous system.

A team of electrical engineers led by Qiuting Huang, a professor at the Institute for Integrated Systems at ETH Zurich, have developed a system that connects implantable LEDs with a tiny device on the subjects head which can be controlled from a tablet via Bluetooth. They have stimulated, with great precision, nerve fibers in the bodies of freely-moving mice, as the scientists report in Nature Biotechnology.

Our goal was to develop an integrated platform as small as possible. Together, the chip, the battery and the antenna for wireless signal transmission weigh less than one gram and occupy less than one cubic centimetre, explained Huang.

While the chip technology enables high integration density of electronic circuits, there are limits to miniaturization, particularly with batteries; the smaller the volume, the greater the energy density. This, in turn, increases the risk that batteries might ignite.

The team originally intended to develop this platform for a project to measure oxygen saturation and blood pressure, but right from the outset of the design phase, they endeavored to ensure the widest possible application for the chip.

Because our system is programmable, we were able to take the electronic circuits that we had been intending to use to measure oxygen saturation in blood and re-purpose them to control the implanted light emitting diodes, said contributing scientist Philipp Schnle.

The group has been collaborating with Stphanie Lacours group at EPFL for five years. Our sophisticated electronics and their soft bioelectronic sensors were made for each other, said Huang. Advances in material sciences and electronics build on each other and interconnect with each other. Together, we have developed an approach that allows us to stimulate a specific nerve fiber in the body of the mouse at precise points in time, added Schnle.

At Harvard Medical School, a research group led by Clifford Woolf wrapped the implants around the sciatic nerve. Without damaging the nerve, over several days, they managed to repeatedly use blue light flashes to activate specific nerve cells known as the nociceptors, which specialize in the transmission of pain signals.

To their surprise, the researchers discovered that repeated optical stimulation of these nociceptors produced a slight reddening in one of the mouses hind paws, a clear sign of inflammation.

Scientists had previously assumed that pain and inflammation were two different processes that arose independently. But now we have been able to prove conclusively that the neurons responsible for pain sensations can also generate an inflammatory immune response, said Woolf. As the researchers explain in the Nature Biotechnology article, these results may potentially point the way to new approaches in such areas as the treatment of chronic pain, or persistent inflammation.

Huang believes that electrical engineering will play an increasingly important role in human health in the future. He highlights the term electroceuticals a combination of electronics and pharmaceuticals which is already being discussed among experts.

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Agios helps identify genes that allow cancer to escape the immune system – FierceBiotech

September 30th, 2020 4:51 pm

Despite the availability of multiple FDA-approved immunotherapies that leverage the bodys immune system to fight cancer, only a fraction of cancer patients are benefiting. That's because cancer cells are cunning. They develop strategies to avoid being targeted and destroyed by immune cells.

To understand the genetic drivers behind the ability of cancer cells to evade the immune system, scientists at the University of Toronto, in collaboration with Agios Pharmaceuticals, used CRISPR to screen six genetically diverse mouse cancer cell lines. They found that genes involved in autophagya process where cells recycle damaged components to regenerate themselveswere key for immune evasion.

The researchers suggested that their findings, published in Nature, open up new venues for the development of immunotherapies that could be effective for largepatient populations across several different tumor types.

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Its very important to understand at the molecular level how cancer develops resistance to immunotherapies in order to make them more broadly available, saidUniversity of Toronto professor Jason Moffat, Ph.D., the studys corresponding author, in a statement.

Moffat and colleagues used CRISPR to screen cancer cells that were cultured in the presence or absence of preactivated killer T cellsimmune cells with a natural ability to hunt and kill cancer. They identified 182 genes that, when deleted, increased either the sensitivity or theresistance of cancer cells to T cells. They called them core cancer-intrinsic cytotoxic T lymphocyte-evasion genes, becauseevery one of them was present across at least three of the six cells lines that the team screened.

The core set of genes included several genes that were known to affect signaling of interferon gamma, a cytokine thats critical to an array of immune responses and that serves as a master communicator with several types of immune cells. Three negative regulators of the interferon-gamma responseSocs1, Ptpn2 and Adaralso emerged as immune-evasion genes.

Several genes identified were related to autophagy. Among the top hits was the Fitm2 gene, which is required for normal fat storage in adipose tissue in mice but was not previously associated with interferon-gamma signaling.

RELATED:New strategies for improving pancreatic cancer treatments

In two mouse models of renal cell carcinoma and melanoma, cancer cells with deleted Fitm2 showed increased cell death after treatment with interferon gamma compared with control cells, the team reported.

Surprisingly, the researchers also found that deleting certain autophagy genes in pairs could make the cells resist T-cell killing. For example, cells that lacked both Atg12 and Atg5 were strongly resistant to the killing effects of T cells as compared with single-mutant cells. This means that if a tumor already harbors a mutation in one autophagy gene, an immunotherapy that targets another autophagy gene could make the disease worse, the researchers explained.

Other oncology researchers are also focusing on the autophagy. A team from the University of Cincinnati, for example, found that mutations in mitochondrial complex I could prevent the autophagy thats triggered by mTOR inhibitors. And researchers at the University of North Carolina reported that targeting the KRAS mutation in pancreatic cancer with an MEK inhibitor made cancer cells more dependent on autophagy for survival. They blocked the process with the anti-malaria drug hydroxychloroquine.

The University of Toronto-led team believes its list of 182 core immune-evasion genes may inform efforts to develop novel cancer immunotherapies. The scientists stressed the need to explorehow combined genetic interactions may alter cancers immune-evasion activity. While simultaneous control of certain genetic features may sensitize cancer cells to immunotherapy, it could make the disease worse in others, they warned.

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Genetic Engineering Drug Market 2020 | What Is The Estimated Market Size In The Upcoming Years? – The Daily Chronicle

September 30th, 2020 4:50 pm

The Global Marketers provides you regional research analysis on Genetic Engineering Drug Market and forecast to 2026. The global Genetic Engineering Drug Market report comprises a valuable bunch of information that enlightens the most imperative sectors of the Genetic Engineering Drug market. The global Genetic Engineering Drug market report provides information regarding all the aspects associated with the market, which includes reviews of the final product, and the key factors influencing or hampering the market growth.

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Main players in the Genetic Engineering Drug Market:

GeneScience Pharmaceuticals Co., LtdBeijing SL Pharmaceutical Co., LtdBiotech Pharmaceutical Co., LtdShenzhen Neptunus Interlong Bio-Technique Co., LtdJiangsu Sihuan Bioengineering Co., LtdTonghua Dongbao Pharmaceutical Co., LtdAnhui Anke Biotechnology (Group) Co., Ltd3SBio Inc.Shanghai Lansheng Guojian Pharmaceutical Co., Ltd

Some of the geographic regions examined in the overall Genetic Engineering Drug Market are:

In addition, the global Genetic Engineering Drug market report delivers brief information about federal regulations and policies that may ultimately affect market growth as well as the financial state. The situation of the global market at the global and regional levels is also described in the global Genetic Engineering Drug market report through geographical segmentation. The Genetic Engineering Drug report introduces speculation attainability evaluation, a task SWOT investigation, and venture yield evaluation.

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Global Genetic Engineering Drug Market Segmentation:

On the Basis of The Application:

30 Years Old30 Years Old-60 Years Old60 Years Old

On the Basis of Type:

Monoclonal AntibodyRecombinant Human ErythropoietinRecombinant Human InterferonRecombinant Human Growth HormoneRecombinant Human Insulin

Moreover, the report comprises the main developments made in the Genetic Engineering Drug market. Porters five force analysis is used to conclude the competition in the Genetic Engineering Drug market along with new entrants and their strategies & tactics. The report involves the value chain analysis which denotes workflow in the Genetic Engineering Drug market. Also, the market has been classified on the basis of category, processes, end-use industry, and region. On the basis of geography, the report Genetic Engineering Drug the market.

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The Genetic Engineering Drug Market research report presents a comprehensive analysis of the market and contains attentive insights, facts, past data, and statistical support, and industry-validated market data. It furthermore contains projections applying a suitable set of assumptions and methodologies. The research Genetic Engineering Drug report provides examination and information according to market segments such as geographies, applications, and industry by considering major players.

Key questions answered in this report

Highlights of the TOC of the Genetic Engineering Drug Report:

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Orphan Drug Exclusivity for CRISPR/Cas-Based Therapeutics – JD Supra

September 30th, 2020 4:50 pm

The prospect of genetic engineering using CRISPR (clustered regularly interspaced short palindromic repeats) and CRISPR-associated nucleases (Cas) has long been hailed as a revolutionary development in medicine.

This technology is rapidly advancing, and several CRISPR/Cas-based drugs have entered clinical trials over the past several years. One kind of product in clinical trials is CRISPR-modified cells, such as CTX001 (CRISPR-Cas9-modified autologous hematopoietic stem cells), currently under study for the treatment of b-thalassemia and severe sickle cell anemia. Another CRISPR-based product, AGN-151587, is injected into the eye with the goal of eliminating a genetic mutation in patients with Leber congenital amaurosis 10, a leading cause of childhood blindness. In parallel, others are working to harness the CRISPR/Cas system to develop drugs for rare diseases, including bespoke therapies tailored to an individual patients needs.

Given CRISPR/Cas-based drugs potential to treat rare diseases, issues relating to orphan drug exclusivity will arise as these products are developed. In May 2020, for example, CTX001 received an orphan drug designation for transfusion-dependent b-thalassemia.

In January 2020, the FDA provided draft guidance regarding orphan drug exclusivity for gene therapy products, which includes CRISPR/Cas gene editing (Draft Guidance). This guidance focuses on the analysis of whether two gene therapy products are the same under the Orphan Drug Act. Although informative, the limited scope of the Draft Guidance invites more questions than it answers.

Same Drugs Under the Orphan Drug Act

Obtaining orphan drug exclusivity involves a two-step process. First, a sponsor requests designation of a drug for a particular rare disease or condition. See 21 C.F.R. 316.20. If this drug is the same drug as a drug already approved to treat the same rare disease or condition, the sponsor must provide a plausible hypothesis that the new drug is clinically superior to the previously-approved drug. Id. Whether two drugs are the same depends on consideration of structural features relevant to that type of drug. See id. 316.3(b)(14).

If the new drug later obtains marketing approval for a use or indication within the rare disease or condition for which it received orphan drug designation, the FDA will determine if the drug is eligible for orphan drug exclusivity. See 21 C.F.R. 316.31(a). In this situation, to receive exclusivity, the sponsor of the new drug must show that its drug is clinically superior to the same previously-approved drug for the same rare disease or condition. See id. 316.34(c). A clinical superiority determination is based on the new drugs greater efficacy, greater safety, or a major contribution to patient care. See id. 316.3(b)(3).

Highlights from Draft FDA Guidance

To determine whether one gene therapy product is the same as another, per 316.3(b)(14)(ii), the FDA will evaluate the principal molecular structural features of the two products, particularly transgenes (e.g., transgenes that encode different enzymes for treatment of the same rare disease) and vectors. For example:

Additionally, [w]hen applicable, the FDA generally intends to consider additional features of the final gene therapy product, such as regulatory elements or, in the case of genetically-modified cells, the type of cell that is transduced. It generally intends to consider requests for designation and exclusivity of gene therapy products to evaluate whether such additional features may also be considered to be principal molecular structural features.

Implications for CRISPR/Cas Therapy Exclusivity

The Draft Guidance helps answer certain high-level questions relating to whether two gene therapy products would be considered the same under the Orphan Drug Act. As various stakeholders have recognized, however, it is short on the details that meaningfully aid the process of drug research and development.

It is clear from the Draft Guidance that a new product can be considered the same as a previously-approved product even if the two products are not perfectly identical, but the guidance does not explain what would constitute a minor difference between such products, or what the scope of additional features would be.

For example, the Draft Guidance does not clarify what makes two transgenes the same. Nor does it cite to prior guidance or regulations that may answer this question. The question is significant because Cas nucleases and other parts of the CRISPR/Cas system may be modified in various ways. To address whether these modifications bar a finding of same-ness, the FDA could potentially import the kinds of considerations that govern same-ness of other kinds of large-molecule products, such as polynucleotide drugs or closely related, complex partly definable drugs with similar therapeutic intent (e.g., viral vaccines). See 21 C.F.R. 316.3(b)(14)(ii)(C), (D). However, this is not clear from the Draft Guidance.

The Draft Guidance also does not explain what will factor into the case-by-case basis assessment of whether viral vectors from the same viral class are the same. In the case of AAV2 and AAV5the two viruses identified in the guidanceresearchers have found that these viruses differ with respect to sequence analysis, tissue tropism, and heparin sensitivity. It is not clear from the guidance, however, whether a plausible hypothesis of clinical superiority will be required to seek orphan drug designation for a drug based on AAV2 if the previously-approved drug expresses the same transgene(s) but is based on AAV5.

It would be beneficial to sponsors and other stakeholders if these aspects of gene therapy drugs sameness are clarified further before they invest significant resources into the design and development of these therapeutics.

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Inventing the future for humankind | Community Perspectives – Fairbanks Daily News-Miner

September 30th, 2020 4:50 pm

Back in the halcyon days, when I somehow got paid for messing with the minds of the impressionable youth of UAF, I liked to ask said minds to project themselves back in time 400 years, to take a look around and report back what, if anything, they noticed different between those times (counting back from now, for example, to the Lords Year 1620. (James I was King, if that helps) and our own times: changes in musical tastes, ethics, physics, theology or attitudes regarding leprosy, for instance.

1620 CE was earlier than heart transplants, genetic engineering and baseball. It was before George Washington and water-seal toilets. Oxygen wouldnt be invented until the mid-1700s (Really: no Periodic Table of Elements, no radioactivity). The Holy Inquisition was in practice: pious religious officials were still torturing heretics and burning witches. It was before abortion rights. Autochthonous peoples in many parts of the world had not been introduced to the blessings of European economics, religion and warfare. It was before Facebook.

Things had changed in the last 400 years. Bigly. My students always got that answer right.

Then Id ask them to project themselves 400 years into the future, to the early 25th Century, say, to look around, to report back. I asked them to pay particular attention to the way our descendants in 2420 look back on our (presumably long-obsolete) ways of doing things: our medicine, say, or our governmental systems, or our responses to global hunger, overpopulation, pollution.

This was a harder task. The problem with prognostication is that we normal people are not particularly good at it, being annoyingly set in our ways. This is not to say that we cant make predictions, but even deeply considered and finely calibrated events such as space launches, brain surgery, or steering an oil tanker around Bligh Reef occasionally go awry. Some events, like nuclear meltdowns or worldwide pandemics, can present unanticipated difficulties.

I asked my students to avoid fantasies like self-aware computers, two-way wrist radios or honest politicians. I was hoping for revolutionary ways of perceiving the world, something on the order of the atalatl, General Relativity or Akira Kurosawa. I was angling for new stuff: examples of true scientific, artistic or musical invention.

My students always protested. Were on to you, old man, you you English teacher! Youve been harping all semester about how we mortals really cant see into the future, that we make up the future with our words. Now you want us to think something no one has ever thought before!

Thats exactly what I wanted them to do, of course. To be fair, really new ideas are not particularly common. It took humans millennia to come up with the atlatl (c.20,000 BCE), even longer to invent the calculus (c.1665 CE) or germ theory (c.1840). But without inventive ways of looking at the world, humanity might still believe that malaria is caused by bad air, that light travels across a medium called luminiferous ether, or that things burn because they contain phlogiston.

Theres been much talk lately of returning to normal, but I wonder if thats really what we want. I wonder if normal isnt what got us into our present public health and economic crises. I think for a lot of people in our community normal is worrying about buying groceries, paying the rent, health care, personal safety.

In this Year of Our Trump and the Corona pandemic (known also to certain elderly cynics as the beer virus or the sniffles) the question for my students would be, Given that we really cant see into the future and given that our current pandemic is unlikely to be our last, whats our best strategy for the survival of Our People (defined however you like) for the next seven generations or so?

Id hope for some inventive thinking along the lines of how to take care of every person on Earth in honest and practical ways. Emphasizing that we have plenty to be humble be about when predicting the future, Id ask them to come up with ideas never tried before. Id suggest that food, shelter and health care need never to be money-dependent, for example. Id ask our youth for creative ways of feeding people, sheltering people, caring for people all people on this, our planetary spaceship.

Id invite them to approach the task with an honest and generous spirit.

Lynn Basham lives in Fairbanks. He taught atthe University of Alaska Fairbanks as an instructor, mostly in the English Department, for about 20 years and retired about10 years ago.

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Family seeks answers, finds hope after daughters diagnosed with rare genetic condition – Steamboat Pilot and Today

September 30th, 2020 4:50 pm

STEAMBOAT SPRINGS You can hear the love in Mariah Gillaspies voice as she talks about her daughters Emma and Abby, who suffer from a rare genetic disease that causes seizures and development issues.

Emma, shes our oldest, and shell be 4 in October, Mariah said. Shes our calm, sweet little child. She has these little coos that sound like a dove. She really enjoys music, and she loves being around other kiddos her age.

Abby is our younger daughter, and shell be 2 in October, and she is our feisty little thing, Mariah continued. So, she lets you know when shes happy; she lets you know when shes not happy.

There is no question the two girls, the only two people in the world believed to have this disease, are surrounded by the love they get from Mariah and their dad Mark.

Mark grew up in Steamboat Springs and graduated from high school here in 2001. The couple now live in Centennial, but Marks parents, Jeanne and Joe Gillaspie, still live in Steamboat as does Marks older brother.

Four years ago, Mark and Mariah were overwhelmed with joy as they welcomed their first child Emma to the world, but when she was three months old, the couple started to notice she was having some strange movements, and when she started having episodes where she would hold her breath until she would turn pale, the couple took her to the doctor.

The doctor initially thought it was reflux, but when Emma stopped breathing in the doctors office, she was rushed to Childrens Hospital of Colorado for more evaluation and tests.

Throughout all this, I was convinced everything was going to be OK, Mariah said. It never crossed my mind that something was seriously wrong, and I had never considered that these were seizures.

Eventually, Emma was diagnosed with infantile spasms, which Mariah said didnt look serious on the outside but were damaging Emmas brain and impacting her development from the inside. Emma started treatment immediately, and the family was encouraged with the results. But then there was a relapse and a new medication, and then another relapse and another new medication.

Mariah said each new medicine came with a longer list of side effects, and Emmas immune system suffered. She had a bout with pneumonia that left her in the hospital for two months.

Through it all, the Gillaspies continued to search for answers.

We did a whole slew of genetic testing, and it came back inconclusive, Mariah said. They found absolutely nothing that could be the cause of her disease, and they told us this is probably some completely random condition that was caused by something that happened in utero.

They also told the Gillaspies that Emmas condition was rare, and there was less than a 3% chance of it happening again. So after extensive genetic testing, they decided to have a second child.

When Abby arrived two years later, they were thrilled, but at about six weeks, they noticed their youngest daughter was displaying the same movements that Emma had shown prior to her diagnosis. So it was back to the doctors, and it was confirmed through genetic testing that Abby and Emma shared the same mutated gene THAP12.

After discovering their daughters were suffering from the same condition, the family embarked on a grassroots effort to drive research about the rare genetic disease, which led to the creation of a foundation, Lightning and Love, a name that was chosen because the family believes lightning struck their family twice in the form of two daughters with the same rare disease.

The doctors would say, Im sorry, theres nothing we can do. We have to wait for science to catch up,' Mariah said. Every doctor that weve encountered has really been amazing and done their very best for us. Its just unfortunate science hasnt caught up to the girls, yet. Thats kind of, whereas parents, were passionate enough to move science along a little faster.

The nonprofit organization is supported by a GoFundMePage, and tax-deductible donations can be made through the Lightning and Love website.

The latest research funded by the foundation involved genetically engineering a zebrafish model to see if it showed symptoms of disease, specifically seizures. The zebrafish did have seizures, which Mariah said was a major breakthrough toward the ultimate goal of finding a gene replacement cure for her daughters.

But the journey for Mark and Mariah has proven to be more than just research and discovery.

What were realizing is the more we talk about it, and the more we do to get our story out there, the more were realizing that theres a lot of other parents that are going through tough times with their kids, too, Mark said. In an odd twist, or an ironic twist, this tough hand that weve been dealt has actually been a very positive light to a lot of other people out there. For me, that is just as important as the research.

The familys story was recently featured on the podcast, Go Shout Love.

The couples positive message is guiding them along the road they hope will lead to a better life for their family. But in the meantime, Mark and Mariah will continue to put smiles ontheir daughters faces the same way most other parents do by offering their love, support and opportunities to find happiness.

For Emma, that means being tossed into the air and caught by her daddy, and for Abby, it is time in her sensory room and being around her dad and her family.

Emma loves very big movements. Shes not mobile, and she cant walk, so when we kind of throw her around in the air or fly her around the room, she absolutely loves it, Mariah said. Abby loves her daddy. She gives big old smiles when he walks into the room.

To reach John F. Russell, call 970-871-4209, email jrussell@SteamboatPilot.com or follow him on Twitter @Framp1966.

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COVID-19 Vaccine and Therapeutics Pipeline Analysis Report 2020: The Race to Market as Clinical Trials Move Up a Gear – ResearchAndMarkets.com -…

September 30th, 2020 4:50 pm

DUBLIN--(BUSINESS WIRE)--The "COVID-19 Vaccine and Therapeutics Pipeline Analysis 2020" report has been added to ResearchAndMarkets.com's offering.

The report covers market characteristics, size and growth, segmentation, regional and country breakdowns, competitive landscape, market shares, trends and strategies for this market. It traces the market's historic and forecast market growth by geography. It places the market within the context of the wider COVID-19 vaccine & therapeutics pipeline analysis 2020 market, and compares it with other markets.

Major players in the COVID-19 vaccine and therapeutics pipeline analysis market are CanSino Biologics, Moderna, Inovio Pharmaceuticals, Regeneron, Gilead Sciences, GlaxoSmithKline, Medicago Inc., Sanofi, University of Oxford, and Altimmune.

The COVID-19 vaccine and therapeutics pipeline analysis market covered in this report is segmented by product type into small molecules, biologics, blood & plasma derivatives, monoclonal antibodies, vaccines, others. It is also segmented by the phase of development into preclinical therapeutics & vaccines, clinical studies, by treatment mechanism & route of administration, and by type of sponsor into pharma/biotech company, academic research/institution, others.

The COVID-19 vaccine and therapeutics pipeline analysis market report provides an analysis of the coronavirus (COVID-19) therapeutics and vaccines under development. The report includes existing vaccines developed against MERS-CoV and SARS-CoV. The novel coronavirus-2019 (nCoV-19) has been named as severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) by the International Committee on Taxonomy of Viruses (ICTV) due to its genetic similarity with the coronavirus responsible for the 2003 SARS outbreak. Currently, government agencies, international health authorities and institutions and biopharmaceutical companies worldwide are focusing on developing vaccines/drugs to prevent or treat the COVID-19 infection.

Ever since the coronavirus hit the world as a global pandemic, many key vaccine developers are collaborating to develop potential COVID-19 vaccine against coronavirus.

Most recently, on 21st May 2020, CanSino Biologics Inc. and Precision NanoSystems announced a co-development agreement of an mRNA lipid nanoparticle (mRNA-LNP) vaccine against COVID-19. The parties will leverage Precision NanoSystems's proprietary RNA vaccine platform, comprising of lipid nanoparticle delivery system and the NanoAssemblr manufacturing technology, to rapidly advance a COVID-19 mRNA-LNP vaccine candidate towards human clinical testing and pursuant to regulatory approvals, and commercialization in different regions. Precision NanoSystems will be responsible for the development of the mRNA-LNP vaccine and CanSinoBIO will be responsible for pre-clinical testing, human clinical trials, regulatory approval and commercialization.

Similarly, on May 19, 2020, IPharmaJet, the maker of innovative, needle-free injection technology announced that its Needle-free Injection System technology will be used to deliver a messenger RNA (mRNA) vaccine against SARS-CoV-2. The vaccine is being developed by Abnova Corporation, the world's largest antibody manufacturer, based in Taiwan.

The development of potential drugs and vaccines for COVID-19 is progressing quickly. There is a massive increase in COVID-19 drugs and vaccines pipeline owing to the urgent need to contain the spread of disease. Government agencies, global health authorities and institutes, and biopharmaceutical companies are focusing on remedies to treat the patients and control the infection spread. Increasing every day, 450+ potential therapeutic candidates are under investigation. While two-thirds of the pipeline account for therapeutic drugs, the remaining one-third accounts for vaccines.

Of the confirmed active vaccine candidates, nearly 70% are being developed by private/industry developers, with the remaining 30% of projects being led by the academic, public sector and other non-profit organizations. Most COVID-19 vaccine development activity is in North America, with around 36 (46%) developers of the confirmed active vaccine candidates. China constitutes 18% with 14 developers, while, Asia excluding China and Europe also constitute 18% each with 14 developers in each region, respectively.

The long and costly drug development process is anticipated to limit the growth of the COVID-19 vaccine & therapeutics. According to the Pharmaceutical Research and Manufacturers of America (PhRMA), the average cost of research and development of a new drug is approximately $2.6 billion. Moreover, the stringent regulations imposed by the various regulatory authorities such as European Medicines Agency and the US Food and Drug Administration (FDA) in regards with clinical trials during the COVID-19 outbreak attributing to the safety of trial participants, maintaining compliance with good clinical practice, and minimizing risks to trial integrity is a major challenge faced by the COVID-19 vaccine and therapeutics market.

The compounds and medications that are under investigation can be grouped into three broad categories - antivirals, immune-system based, and vaccines. The anti-virals including Darunavir, Favipiravir, Hydroxychloroquine and chloroquine, Lopinavir, and Remdesivir (GS-5734), immune system-related therapies including Tocilizumab, Tocilizumab, and Vitamin C, and other medications are currently being evaluated as therapies. Three key drugs are currently in phase III, of which are two small molecule-based drugs, Remdesivir by Gilead Sciences Inc. and Favipiravir by Fujifilm Toyama Chemical Co Ltd, and Sarilumab, a monoclonal antibody by Regeneron Pharmaceutical. With regards to the prophylactic vaccine pipeline, more than 90% are in early-stage development (discovery and preclinical), and only three in Phase II. These three COVID-19 vaccines are being developed by Sinovac Biotech Ltd, the University of Oxford, and the third vaccine, named CIGB-2020, is being developed by the Center for Genetic Engineering and Biotechnology.

According to the European Centre for Disease Prevention and Control, worldwide, there are over 10.8 million cases of COVID-19. Globally, R&D spending has increased to find a potential drug or vaccine to combat this pandemic. Currently, there is no approved targeted therapy for patients with COVID-19. However, an array of drugs approved for other indications as well as several new investigational drugs are being studied in several hundred clinical trials. The increased R&D spending has contributed to the invention/discovery of more than 400 unique drugs to treat COVID-19 among which 298 are therapeutic drugs and 140 prophylactic vaccines that are spread across all stages of development (Discovery, Preclinical, Phase I, Phase II, and Phase III). As of June 2020, over 2,341 clinical trials are investigating potential therapies for COVID-19, of which nearly 800 are interventional trials.

Other Collaborations:

Key Topics Covered:

1. Executive Summary

2. Disease Overview

2.1. Novel Coronavirus Etiology and Pathogenesis

2.2. Novel Human Coronavirus (ClOVID-19) Clinical Features-Signs and Symptoms

3. Disease Epidemiology and Epidemic Statistics for Major countries

4. Global Pipeline Analysis of COVID-19 Therapeutics and Vaccines

4.1 Global Pipeline Analysis, By Product Type

4.1.1 Small Molecules

4.1.2 Biologics

4.1.2.1 Blood & Plasma Derivatives

4.1.2.2 Monoclonal Antibodies

4.1.2.3 Vaccines

4.1.2.4 Others

4.2 Global Pipeline Analysis, By Phase of development

4.2.1 Preclinical Therapeutics & Vaccines

4.2.2 Clinical Studies

4.2.2.1 Clinical Phase I, II, III

4.3 Global Pipeline Analysis, By Treatment Mechanism & Route of Administration

4.3.1 Mechanism of Action

4.3.1.1 Viral Replication Inhibitors

4.3.1.2 Protease Inhibitors

4.3.1.3 Immunostimulants

4.3.1.4 Other Mechanism of Action

4.3.2 Route of Administration

4.3.2.1 Oral

4.3.2.2 Intravenous

4.3.2.3 Subcutaneous

4.3.2.4 Other Route of Administration

4.4 Global Pipeline Analysis, By Type of Sponsor

4.4.1 Pharma/Biotech Company

4.4.2 Academic Research/Institution

4.4.3 Others such as Government Organizations and CROs

5. Competitive Landscape for Late Stage Therapeutics and Vaccine

5.1 Company Overview

5.2 Product Description

5.3 Research and Development

5.3.1 Non-Clinical Studies

5.3.2 Clinical Studies

5.3.3 Highest/Late Stage Development Activities

5.4 Licensing and Collaboration Agreements

5.5 Milestones & Future Plans

6. Regulatory Framework for COVID-19 Therapeutics and Vaccines Marketing Approvals

6.1 Regulatory Framework in the USA

6.2 Regulatory Framework in EU and Other Countries

7. Recommendations & Conclusion

Companies Mentioned

For more information about this report visit https://www.researchandmarkets.com/r/yg3jj7

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