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FDA Continues Strong Support of Innovation in Development of Gene Therapy Products – MyChesCo

February 1st, 2020 4:41 am

WASHINGTON, D.C. This is a pivotal time in the field of gene therapy as the FDA continues its efforts to support innovators developing new medical products for Americans and others around the world. To date, the FDA has approved four gene therapy products, which insert new genetic material into a patients cells.

The agency anticipates many more approvals in the coming years, as evidenced by the more than 900 investigational new drug (IND) applications for ongoing clinical studies in this area. The FDA believes this will provide patients and providers with increased therapeutic choices.

In that spirit, the FDA announced the release of a number of important policies: six final guidances on gene therapy manufacturing and clinical development of products and a draft guidance, Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations.

The growth of innovative research and product development in the field of gene therapy is exciting to us as physicians, scientists and regulators, said FDA Commissioner Stephen M. Hahn, M.D. We understand and appreciate the tremendous impact that gene therapies can have on patients by potentially reversing the debilitating trajectory of diseases. These therapies, once only conceptual, are rapidly becoming a therapeutic reality for an increasing number of patients with a wide range of diseases, including rare genetic disorders and autoimmune diseases.

As the regulators of these novel therapies, we know that the framework we construct for product development and review will set the stage for continued advancement of this cutting-edge field and further enable innovators to safely develop effective therapies for many diseases with unmet medical needs, said Peter Marks, M.D., Ph.D., director of the FDAs Center for Biologics Evaluation and Research. Scientific development in this area is fast-paced, complex, and poses many unique questions during a product review; including how these products work, how to administer them safely, and whether they will continue to achieve a therapeutic effect in the body without causing adverse side effects over a long period of time.

One of the most important steps the FDA can take to support safe innovation in this field is to create policies that provide product developers with meaningful guidance to answer critical questions as they research and design their gene therapy products.

The six final guidances issued today provide the agencys recommendations for product developers on manufacturing issues and recommendations for those focusing on gene therapy products to address specific disease areas.

The six guidance documents incorporate input from many stakeholders and take a significant step toward helping to shape the modern structure for the development and manufacture of gene therapies.

The agency is issuing this suite of documents to help advance the field of gene therapy while providing recommendations to help ensure that these innovative products meet the FDAs standards for safety and effectiveness.

The scientific review of gene therapies includes the need to evaluate highly complex information on product manufacturing and quality. In addition, the clinical review of these products frequently poses more challenging questions to regulators than reviews of more conventional drugs, such as questions about the durability of response, and these questions often cant be fully answered in pre-market trials of reasonable size and duration.

For some gene therapy products, therefore, although they have met the FDAs standards for approval, the agency may need to accept some level of uncertainty around questions of the duration of the response at the time of marketing authorization.

Effective tools for reliable post-market follow up, such as post-market clinical trials, are going to be key to advancing this field and helping to ensure that the agencys approach fosters safe and innovative treatments.

The draft guidance on interpreting sameness of gene therapy products under the orphan drug regulations provides the FDAs proposed current thinking on an interpretation of sameness between gene therapy products for the purposes of obtaining orphan-drug designation and eligibility for orphan-drug exclusivity.

The draft guidance focuses on how the FDA will evaluate differences between gene therapy products when they are intended to treat the same disease. As laid out in the FDAs draft guidance and regulations, the agencys determination will consider the principal molecular structural features of the gene therapy products, which includes transgenes (the transferred gene) and vectors (the vehicle for delivering the transgene to a cell).

With the large volume of products currently being studied, gene therapy product developers have asked the agency important questions about orphan-drug designation incentives to develop products for rare diseases with very small patient populations.

The draft guidance has potential positive implications both for product developers and patients by providing insight into the agencys most current thinking on the sameness of products, and thus, not discourage the development of multiple gene therapy products to treat the same disease or condition.

For patients, this policy could help lead to the development and approval of multiple treatments, creating a more competitive market with choices. The FDA encourages stakeholders to provide their comments.

In sum, these policy documents are representative of efforts to help advance product development in the field of gene therapy. The FDA will continue to work with product innovators, sponsors, researchers, patients, and other stakeholders to help make the development and review of these products more efficient, while putting in place the regulatory controls needed to ensure that the resulting therapies are both safe and effective.

The agency also encourages developers of new gene therapy products to make full use of FDAs expedited programs available for products intended to address unmet medical needs in the treatment of serious or life-threatening conditions.

These programs include breakthrough therapy designation, regenerative medicine advanced therapy designation, and fast track designation, as well as priority review and accelerated approval. Developers should pursue these programs whenever possible to help bring the benefits of important advances to patients as soon as possible.

The FDA believes their work will help advance innovations in a way that assures their safety and effectiveness, provides new therapeutic choices to patients and providers and continues to build confidence in this novel and emerging area of medicine.

Source: Food and Drug Administration

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Cedars-Sinai Study Indicates That Parkinson’s Disease May Start Before Birth – Equities.com

February 1st, 2020 4:41 am

Image: Nur Yucer, PhD, a project scientist, and Clive Svendsen, PhD, director of the Cedars-Sinai Board of Governors Regenerative Medicine Institute and Professor of Biomedical Sciences and Medicine at Cedars-Sinai. Photo by Cedars-Sinai.

Parkinson's disease is a neurodegenerative disorder that affects predominately dopamine-producing neurons in the brain. Nearly one million will be living with Parkinson's disease in the US this year, according to the Parkinson's Foundation. This is more than the number of people diagnosed with multiple sclerosis, muscular dystrophy and Lou Gehrig's diseasecombined.

About 60,000 Americans are diagnosed with Parkinson's disease each year, and more than 10 million people worldwide are living with it. Incidence of Parkinsons disease increases with age, but an estimated 10 percent of people with Parkinson's disease are diagnosed before age 50. This is called young-onset Parkinson's.

Researchers at Cedars-Sinai, led by Clive Svendsen, PhD, director of the Cedars-Sinai Board of Governors Regenerative Medicine Institute and Professor of Biomedical Sciences and Medicine at Cedars-Sinai, reported in a study published in Nature Medicine that they found that patients who develop young-onset Parkinsons disease may have been born with dysfunctional brain cells that go undetected for decades.

The research team generated special stem cells, known as induced pluripotent stem cells (iPSCs), from cells of patients suffering from young-onset Parkinsons disease. These iPSCswhich can produce any cell type of the human body, all genetically identical to the patients own cellswere used to produce dopamine neurons from each patient to analyze their functions.

Two key abnormalities were observed in these neurons:

- Dr. Clive Svendsen

After testing a number of drugs on the abnormal dopamine neurons, the researchers discovered that a drug called PEP005 (ingenol mebutate) reduced the elevated levels of alpha-synuclein in both the dopamine neurons in the dish and in laboratory mice. A gel formulation of PEP005 is marketed by LEO Pharma as Picato and is FDA-approved for the treatment of actinic keratosis, a scaly skin patch that develops from years of exposure to the sun. According to the Mayo Clinic, a small percentage of actinic keratosis lesions can eventually become skin cancer.

Michele Tagliati, PhD, Director of the Movement Disorders Program and Vice Chair and Professor in the Department of Neurology at Cedars-Sinai, said the research team next will study how PEP005 might be delivered to the brain and whether or not the abnormalities found in young-onset Parkinson's patients also exist in other forms of Parkinsons.

- Dr. Michele Tagliati.

Edward Kim is Managing Editor of Equities.com.

_____

Sources: Equities News, Cedars-Sinai

DISCLOSURE:The views and opinions expressed in this article are those of the authors, and do not represent the views of equities.com. Readers should not consider statements made by the author as formal recommendations and should consult their financial advisor before making any investment decisions. To read our full disclosure, please go to: http://www.equities.com/disclaimer.

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Nanomedicine Market: Industry Analysis and forecast 2026 – Expedition 99

January 30th, 2020 3:46 pm

Nanomedicine Marketwas valued US$ XX Bn in 2018 and is expected to reach US$ XX Bn by 2026, at CAGR of XX% during forecast period of 2019 to 2026.

Nanomedicine Market Drivers and Restrains:Nanomedicine is an application of nanotechnology, which are used in diagnosis, treatment, monitoring, and control of biological systems. Nanomedicine usages nanoscale manipulation of materials to improve medicine delivery. Therefore, nanomedicine has facilitated the treatment against various diseases. The nanomedicine market includes products that are nanoformulations of the existing drugs and new drugs or are nanobiomaterials. The research and development of new devices as well as the diagnostics will become, more effective, enabling faster response and the ability to treat new diseases are likely to boost the market growth.

The nanomedicine markets are driven by factors such as developing new technologies for drug delivery, increase acceptance of nanomedicine across varied applications, rise in government support and funding, the growing need for therapies that have fewer side effects and cost-effective. However, long approval process and risks associated with nanomedicine (environmental impacts) are hampering the market growth at the global level. An increase in the out-licensing of nanodrugs and growth of healthcare facilities in emerging economies are likely to create lucrative opportunities in the nanomedicine market.

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Nanomedicine Market Segmentation Analysis:Based on the application, the nanomedicine market has been segmented into cardiovascular, neurology, anti-infective, anti-inflammatory, and oncology. The oncology segment held the dominant market share in 2018 and is projected to maintain its leading position throughout the forecast period owing to the rising availability of patient information and technological advancements. However, the cardiovascular and neurology segment is projected to grow at the highest CAGR of XX% during the forecast period due to presence of opportunities such as demand for specific therapeutic nanovectors, nanostructured stents, and implants for tissue regeneration.

Nanomedicine Market Regional Analysis:Geographically, the Nanomedicine market has been segmented into North America, the Europe, Asia Pacific, Latin America, and Middle East & Africa. North America held the largest share of the Nanomedicine market in 2018 due to the rising presence of patented nanomedicine products, the availability of advanced healthcare infrastructure and the rapid acceptance of nanomedicine. The market in Asia Pacific is expected to expand at a high CAGR of XX% during the forecast period thanks to rise in number of research grants and increase in demand for prophylaxis of life-threatening diseases. Moreover, the rising investments in research and development activities for the introduction of advanced therapies and drugs are predicted to accelerate the growth of this region in the near future.

Nanomedicine Market Competitive landscapeMajor Key players operating in this market are Abbott Laboratories, CombiMatrix Corporation, General Electric Company, Sigma-Tau Pharmaceuticals, Inc, and Johnson & Johnson. Manufacturers in the nanomedicine are focusing on competitive pricing as the strategy to capture significant market share. Moreover, strategic mergers and acquisitions and technological innovations are also the key focus areas of the manufacturers.

The objective of the report is to present a comprehensive analysis of Nanomedicine Market including all the stakeholders of the industry. The past and current status of the industry with forecasted market size and trends are presented in the report with the analysis of complicated data in simple language. The report covers all aspects of the industry with a dedicated study of key players that includes market leaders, followers and new entrants by region. PORTER, SVOR, PESTEL analysis with the potential impact of micro-economic factors by region on the market are presented in the report. External as well as internal factors that are supposed to affect the business positively or negatively have been analyzed, which will give a clear futuristic view of the industry to the decision-makers. The report also helps in understanding Nanomedicine Market dynamics, structure by analyzing the market segments and project the Nanomedicine Market size. Clear representation of competitive analysis of key players By Type, Price, Financial position, Product portfolio, Growth strategies, and regional presence in the Nanomedicine Market make the report investors guide.

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Scope of the Nanomedicine Market:

by Modality:

Diagnostics Treatmentsby Diseases:

Oncological Diseases Infectious Diseases Cardiovascular Diseases Orthopedic Disorders Neurological Diseases Urological Diseases Ophthalmological Diseases Immunological Diseases

by Application:

Neurology Cardiovascular Anti-Inflammatory Anti-Infectives Oncology

by Region:

Asia Pacific North America Europe Latin America Middle East Africa

Major Players:

Abbott Laboratories CombiMatrix Corporation General Electric Company Sigma-Tau Pharmaceuticals, Inc Johnson & Johnson Mallinckrodt plc. Merck & Company, Inc. Nanosphere, Inc. Pfizer, Inc. Teva Pharmaceutical Industries Ltd. Celgene Corporation UCB (Union Chimique Belge) S.A. AMAG Pharmaceuticals Nanospectra Biosciences, Inc. Arrowhead Pharmaceuticals, Inc. Leadiant Biosciences, Inc. Epeius Biotechnologies Corporation Cytimmune Sciences, Inc.

Browse Full Report with Facts and Figures of Nanomedicine Market Report at:https://www.maximizemarketresearch.com/market-report/nanomedicine-market/39223/

MAJOR TOC OF THE REPORT

Chapter One: Nanomedicine Market Overview

Chapter Two: Manufacturers Profiles

Chapter Three: Global Nanomedicine Market Competition, by Players

Chapter Four: Global Nanomedicine Market Size by Regions

Chapter Five: North America Nanomedicine Revenue by Countries

Chapter Six: Europe Nanomedicine Revenue by Countries

Chapter Seven: Asia-Pacific Nanomedicine Revenue by Countries

Chapter Eight: South America Nanomedicine Revenue by Countries

Chapter Nine: Middle East and Africa Revenue Nanomedicine by Countries

Chapter Ten: Global Nanomedicine Market Segment by Type

Chapter Eleven: Global Nanomedicine Market Segment by Application

Chapter Twelve: Global Nanomedicine Market Size Forecast (2019-2026)

About Us:

Maximize Market Research provides B2B and B2C market research on 20,000 high growth emerging technologies & opportunities in Chemical, Healthcare, Pharmaceuticals, Electronics & Communications, Internet of Things, Food and Beverages, Aerospace and Defense and other manufacturing sectors.

Contact info:

Name: Vikas Godage

Organization: MAXIMIZE MARKET RESEARCH PVT. LTD.

Email: sales@maximizemarketresearch.com

Contact: +919607065656/ +919607195908

Website:www.maximizemarketresearch.com

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These 4 Biotech Stocks Are the Silver Lining on the Wuhan Coronavirus – Yahoo Finance

January 30th, 2020 3:46 pm

With the outbreak of the Wuhan coronavirus in China and the increasing death toll, there is little doubt that global financial markets are going to be impacted negatively. As Trump considers banning all flights to China, airline and travel companies like United Airlines (NASDAQ:UAL), Expedia (NASDAQ:EXPE), American Airlines (NASDAQ:AAL) and many more have seen their stock prices crash. There is a strong negative sentiment on the Chinese economy as a whole as the virus has shaken up many of the erstwhile strong sectors.

Overview of the virus

For those readers who are not aware, the Wuhan coronavirus first emerged in the central Chinese city of the same name through the meat and seafood markets and has spread across the country with over a thousand cases and more than a hundred deaths confirmed. It belongs to the same family of viruses as SARS (severe acute respiratory syndrome) and the MERS (Middle East respiratory syndrome). This group of crown-shaped viruses can become deadly if it causes the patient to develop lower respiratory tract illnesses such as pneumonia or bronchitis. It is highly contagious in nature, spreading through the slightest form of saliva contact, whether it is coughing or kissing. The story of the Wuhan coronavirus sounds like history repeating itself after SARS shook up global markets in 2002-03.

It is worth recalling that pharma and biotech companies catering to the respiratory system and providing anti-viral medications were the ones that appreciated the most during the time of SARS, and the current situation does not appear very different. With a strong negative sentiment prevailing in most other sectors, pharma and biotech are perhaps the only sectors that could get a boost from a new bullish sentiment resulting from this virus. Based on the nature of the virus and the expected treatments, the following four stocks could benefit hugely given their presence in the field of anti-viral respiratory medication.

GlaxoSmithKline plc

Since the Wuhan coronavirus belongs to the same family as the SARS, it is important to recall those companies which benefitted the most from the SARS outbreak. While SARS may not have a defined cure even today, the most commonly prescribed form of treatment is the same as that for pneumonia. GlaxoSmithKline plc (NYSE:GSK) is one of the leaders in the space of anti-viral treatments for respiratory disorders like pneumonia.

The above chart shows how the company's stock grew as much as 35% during the SARS phase, which is quite significant for its size. It is worth highlighting that GlaxoSmithKline is a global player with its biggest markets being the U.S. and the UK. Respiratory oral health is one of its strongest segments, and it has a monopolistic position in many anti-viral medications. For example, its Shingrix vaccine for shingles is the only preventive vaccine for the disease across the globe. It has also performed strong research on HIV. The stock has appreciated by over 20% in the past twelve months and also provided a dividend yield of 4.32%, making it an excellent bet for investors.

Abbott Laboratories

Abbott Laboratories (NYSE:ABT) is more of a play on the diagnostics aspect rather than the treatment aspect of the Wuhan virus. The company is one of the largest global biotech giants, and one of its most important offerings relevant to the Wuhan virus situation is its diagnostic capabilities. Abbott is known to provide rapid diagnostics systems for infectious diseases along with remote patient monitoring, informatics and automation solutions that are all very relevant to diagnose the Wuhan virus victims. It also has molecular point-of-care testing for HIV, influenza A and B and RSV.

Story continues

Abbott's relevance was so strong in the SARS era that it appreciated by over 30% in those times and is already up by around 22% in the past 12 months. Given the current situation, the upward momentum of the stock might continue for a while.

Gilead Sciences, Inc.

Biotech giant Gilead Sciences, Inc. (NASDAQ:GILD) was easily one of the biggest beneficiaries in the SARS outbreak, as it saw its stock appreciate more than 200% throughout the outbreak.

The reason for this appreciation is that Gilead gets most of its revenues from the anti-viral segment. The company's stock price has been stable throughout economic downturns and it is not without reason that the company has a 4.5-star business predictability rating on Gurufocus. Not only is it debt-free, it is known to distribute a good amount of dividend (current yield of 3.93%) to shareholders over and above capital appreciation. Not only has it done remarkable research on HIV, Gilead also has some very well known anti-viral brands in the market such as Atripla, Cayston, Sovaldi, Odefsey, Truvada, Biktarvy and so on. It is certainly going to be moving fast in the race to provide strong anti-viral treatments for the Wuhan coronavirus.

NanoViricides, Inc.

NanoViricides, Inc. (NNVC) is the only small, development-stage company on this list, but it is here for a reason. Since the news of the Wuhan virus outbreak, NanoViricides has seen its stock shoot up by as much as 349%.

The company, led by biotech veteran Dr. Anil Diwan, specializes in anti-viral research and had actively worked on MERS in the past. Its current research is also focused on treating viruses through its proprietary nanomedicine technology, where it uses anti-viral nanomachines known as "nanoviricides." The company has a decent pipeline of anti-viral drug candidates catering to diseases such as shingles, herpes, seasonal and potentially-epidemic influenzas, bird flu, HIV, cold sores, viral eye diseases and dengue viruses.

In fact, its most rapidly advancing drug candidate is a topical cream for the treatment of shingles, which is now advancing to the stage of IND application before progressing to human trials. While the company may not have significant revenues today, it hopes to start monetizing the progress of this cream through licensing agreements after the initial phases of the human trials. It has been one of the biggest beneficiaries of the Wuhan virus outbreak so far in terms of stock appreciation, and the upward momentum is expected to remain strong with the IND application results arriving soon.

Conclusion

The Wuhan coronavirus may have a huge negative impact on global markets over a span of time, but the pharma and biotech space is one where it presents a good opportunity. As its fears grip the world, companies like the ones mentioned above are working hard to capitalize on this opportunity and maximize their revenues. In such a situation, it is often beneficial for investors to be opportunistic and make the most returns through investing in these companies.

Disclosure: No positions.

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Global Nanorobotics Market : Industry Analysis and Forecast (2018-2026) – Expedition 99

January 30th, 2020 3:46 pm

Global Nanorobotics Marketwas valued at US$ 3.7 Bn in 2017 and is expected to reach US$ 9.2Bn by 2026, at a CAGR of 12.06%during a forecast period.Global Nanorobotics MarketDevelopments in nanotechnology coupled with demand for minimally aggressive procedures are expected to drive market growth over the forecast period. Nanobots possess likely in the medical sector for destroying cancerous cells at the genetic level. Increasing support for nanomedicine by many nations and the increasing geriatric population are factors which can augur market demand.

Utilization of nanobots in the ranostics can be beneficial for the market in the near future. A rise in miniaturization and demand for automation across various sectors are anticipated to fuel market growth. Training of new personnel to use nanobots can restrain market growth in the upcoming years.Nanomedicine application segment to grow at the highest CAGR during the forecast period. Nanorobotics is widely used in nanomedicine owning to its healthcare features. The large share of this application aspects to the large level of commercialization in the healthcare sector for drug delivery, in vivo imaging, biomaterial, in vitro diagnostic, active implants, and drug therapy.

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North America region accounted for the largest share of 12.2%, in terms of value, of the nanorobotics market globally. Presence of many nanotechnology companies, well-developed healthcare infrastructure, and government initiatives to create patient awareness are factors driving the market. The U.S is anticipated to contribute to market revenue owing to the increase in cardiovascular diseases and the rising elderly populace.

Europe follows North America as the second biggest nanorobotics market. Presence of chronic diseases and the burgeoning population are factors expected to indicate the Europe nanobots market. Establishment of organizations to develop standards pertaining to nanotechnology can expand market growth. In 2018, DNA-Robotics, an organization including 12 European companies, has outlined steps to expedite production of nanobots on a large scale. These standards can help scale the market exponentially in the upcoming years.

A recent development in nanorobotics market: In March 2018, Thermo Fisher Scientific acquired Gatan, an exclusively owned subsidiary of Roper Technologies. Gatan is an electron microscopy solutions provider in the U.S, which accompaniments the Thermo Fisher Scientifics electron microscopy solutions business.In March 2017, Oxford Instruments (U.K) Asylum Research introduced its new SurfRider HQ-Series of high quality, budget-priced AFM probes, which are also existing in a model suitable for nanomechanical image mode.

The objective of the report is to present a comprehensive assessment of the market and contains thoughtful insights, facts, historical data, industry-validated market data and projections with a suitable set of assumptions and methodology. The report also helps in understanding Global Nanorobotics Market dynamics, structure by identifying and analyzing the market segments and project the global market size. Further, the report also focuses on the competitive analysis of key players by product, price, financial position, product portfolio, growth strategies, and regional presence. The report also provides PEST analysis, PORTERs analysis, SWOT analysis to address the question of shareholders to prioritizing the efforts and investment in the near future to the emerging segment in the Global Nanorobotics Market.

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Scope of the Global Nanorobotics Market

Global Nanorobotics Market, By Type

Nanomanipulatoro Electron Microscope (EM) Scanning Electron Microscope (SEM) Transmission Electron Microscope (TEM)o Scanning Probe Microscope (SPM) Atomic Force Microscopes (AFM) Scanning Tunneling Microscope (STM) Bio-Nanorobotics Magnetically Guided Bacteria-BasedGlobal Nanorobotics Market, By Application

Nanomedicine Biomedical Mechanical OthersGlobal Nanorobotics Market, By Region

North America Europe Asia Pacific Middle East and Africa South AmericaKey players operating in Global Nanorobotics Market:

Bruker JEOL Thermo Fisher Scientific Ginkgo Bioworks Oxford Instruments EV Group Imina Technologies Toronto Nano Instrumentation KlockeNanotechnik KleindiekNanotechnik Xidex Synthace Park Systems Smaract Nanonics ImagingKey Innovators:

Novascan Technologies Angstrom Advanced Hummingbird Scientific NT-MDT Spectrum Instruments Witec

Browse Full Report with Facts and Figures of Nanorobotics Market Report at:https://www.maximizemarketresearch.com/market-report/global-nanorobotics-market/30888/

MAJOR TOC OF THE REPORT

Chapter One: Nanorobotics Market Overview

Chapter Two: Manufacturers Profiles

Chapter Three: Global Nanorobotics Market Competition, by Players

Chapter Four: Global Nanorobotics Market Size by Regions

Chapter Five: North America Nanorobotics Revenue by Countries

Chapter Six: Europe Nanorobotics Revenue by Countries

Chapter Seven: Asia-Pacific Nanorobotics Revenue by Countries

Chapter Eight: South America Nanorobotics Revenue by Countries

Chapter Nine: Middle East and Africa Revenue Nanorobotics by Countries

Chapter Ten: Global Nanorobotics Market Segment by Type

Chapter Eleven: Global Nanorobotics Market Segment by Application

Chapter Twelve: Global Nanorobotics Market Size Forecast (2019-2026)

About Us:

Maximize Market Research provides B2B and B2C market research on 20,000 high growth emerging technologies & opportunities in Chemical, Healthcare, Pharmaceuticals, Electronics & Communications, Internet of Things, Food and Beverages, Aerospace and Defense and other manufacturing sectors.

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Organization: MAXIMIZE MARKET RESEARCH PVT. LTD.

Email: sales@maximizemarketresearch.com

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Is GeneReach Biotechnology Corp. (GTSM:4171) Investing Effectively In Its Business? – Simply Wall St

January 30th, 2020 3:45 pm

Today well evaluate GeneReach Biotechnology Corp. (GTSM:4171) to determine whether it could have potential as an investment idea. To be precise, well consider its Return On Capital Employed (ROCE), as that will inform our view of the quality of the business.

First of all, well work out how to calculate ROCE. Second, well look at its ROCE compared to similar companies. Then well determine how its current liabilities are affecting its ROCE.

ROCE measures the return (pre-tax profit) a company generates from capital employed in its business. In general, businesses with a higher ROCE are usually better quality. Overall, it is a valuable metric that has its flaws. Renowned investment researcher Michael Mauboussin has suggested that a high ROCE can indicate that one dollar invested in the company generates value of more than one dollar.

Analysts use this formula to calculate return on capital employed:

Return on Capital Employed = Earnings Before Interest and Tax (EBIT) (Total Assets Current Liabilities)

Or for GeneReach Biotechnology:

0.11 = NT$72m (NT$713m NT$61m) (Based on the trailing twelve months to September 2019.)

Therefore, GeneReach Biotechnology has an ROCE of 11%.

View our latest analysis for GeneReach Biotechnology

One way to assess ROCE is to compare similar companies. Using our data, GeneReach Biotechnologys ROCE appears to be around the 9.5% average of the Medical Equipment industry. Regardless of where GeneReach Biotechnology sits next to its industry, its ROCE in absolute terms appears satisfactory, and this company could be worth a closer look.

GeneReach Biotechnology has an ROCE of 11%, but it didnt have an ROCE 3 years ago, since it was unprofitable. That implies the business has been improving. The image below shows how GeneReach Biotechnologys ROCE compares to its industry, and you can click it to see more detail on its past growth.

When considering this metric, keep in mind that it is backwards looking, and not necessarily predictive. ROCE can be misleading for companies in cyclical industries, with returns looking impressive during the boom times, but very weak during the busts. ROCE is only a point-in-time measure. Since the future is so important for investors, you should check out our free report on analyst forecasts for GeneReach Biotechnology.

Current liabilities are short term bills and invoices that need to be paid in 12 months or less. Due to the way the ROCE equation works, having large bills due in the near term can make it look as though a company has less capital employed, and thus a higher ROCE than usual. To counteract this, we check if a company has high current liabilities, relative to its total assets.

GeneReach Biotechnology has total assets of NT$713m and current liabilities of NT$61m. As a result, its current liabilities are equal to approximately 8.6% of its total assets. With low current liabilities, GeneReach Biotechnologys decent ROCE looks that much more respectable.

This is good to see, and while better prospects may exist, GeneReach Biotechnology seems worth researching further. GeneReach Biotechnology shapes up well under this analysis, but it is far from the only business delivering excellent numbers . You might also want to check this free collection of companies delivering excellent earnings growth.

If you like to buy stocks alongside management, then you might just love this free list of companies. (Hint: insiders have been buying them).

If you spot an error that warrants correction, please contact the editor at editorial-team@simplywallst.com. This article by Simply Wall St is general in nature. It does not constitute a recommendation to buy or sell any stock, and does not take account of your objectives, or your financial situation. Simply Wall St has no position in the stocks mentioned.

We aim to bring you long-term focused research analysis driven by fundamental data. Note that our analysis may not factor in the latest price-sensitive company announcements or qualitative material. Thank you for reading.

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A case of wholehearted biotechnology adoption – The Hindu

January 30th, 2020 3:45 pm

In the Editorial page article, The flawed spin to Indias cotton story (January 23, 2020), there are unfounded claims about a technology that has in reality been a boon to farmers across the world.

The first point made in the article is that GM cotton covers 95% of the area under cotton and that there are no choices for farmers. The fact: Indian farmers have voted for choice of seeds with biotechnologies by planting hybrid cotton biotech seeds on over 90% of the countrys cotton acreage. They want seeds and technologies that provide optimal yield, income and convenience in cultivation. Today, they choose from over 800 hybrid Bt cotton seed brands from over 40 Indian and global seed companies, with five approved in-the-seed insect protection Bt cotton technologies and non-Bt varietal cotton seeds. Farmers have not shown any preference for planting non-Bt cotton seeds including the quantity supplied along with the Bt cotton seed by seed companies as per regulatory guidelines.

Several key studies by third-party economists and sociologists have established that 85% of hybrid Bt cotton seed farmers and farm labourers invested in better education for children; 77% reported better intake of nutritious food; 75% reported better health of their family members; 64% invested on the health of livestock; female workers on Bt cotton fields earned an average 55% higher income; and 42.4 crore additional days of rural employment have been generated, thereby doubling cotton production.

Indias farmers are the ones who have reposed trust in biotechnology, making India the worlds second largest cotton producer and exporter by doubling cotton production over the past decade.

Cotton Corporation of India data show that the highest production of 398 lakh bales of cotton in India was achieved in 2013-14, valued at around 72,000 crore. Additional incomes were generated from cotton seeds oil (1.3 million tons) and cotton seed oilmeal (11 million tons) worth 13,000 crore and 22,000 crore, respectively. The Bt cotton seed market is about 3,000 crore, making it hardly 2.5% of the total value generated.

The articles second point is about low productivity as compared to the global scene. The fact is that technology has not only increased yields but also greatly reduced pesticide use. Biotechnology in cotton, post its introduction in 2002, has led to transformational changes in Indias cotton cultivation. These have helped increase cotton yields by over 1.8 times from 241 kg/hectare in 2002-2003 to 541 kg/hectare in 2018-2019. A BKS-CSD study shows that the significant increase in farmer incomes from higher yields and reduced pesticide use has generated additional farm income of over 42,300 crore. India is moving to first place as the largest producer of cotton in the world.

However, it is not just the technology that increases yields. Indias farmers face numerous uncertainties and crop management challenges, affecting farm yield and incomes; knowledge of cultivation and correct agronomic practices can make a significant impact. This is being addressed by numerous extension efforts.

There is an opportunity to increase yields further in India when compared to other countries that have been using even more advanced GM traits than what is being used in India. New technology introduction has stopped in India since 2005, affecting growth of yields.

The articles third point is about the availability of low cost manual labour. The fact is that one of the major challenges lies in securing labour to conduct field operations. Today, labour accounts for over 58% of a farmers cost of cultivation per acre. In a fast-evolving global market, Indias farmers instead need the best technologies to remain competitive.

The next claim is about varieties offering farmers increased benefits than hybrid cotton seeds. The fact is that Indian farmers who were using varieties for years switched to hybrids in the mid-1980s mainly because of the enormous benefits. Cotton Advisory Board data show that Indias cotton yields which were at 169 kg/hectare in 1980-81 increased to 278 kg/hectare in 2000-01 and then 542 kg/ hectare in 2016-17.

The writers argument that High Density Planting (HDP) took place in various countries after introduction of biotech cotton is inaccurate. Planting rates are determined by several agronomic and environmental conditions and not based on biotech versus non-biotech. There is also no change in the seed rate in any of the countries in which biotech cotton has been adopted. HDP has done well in India because of the better quality of germplasm.

Also, Turkey is not a large cotton producer. USDA statistics in 2017-18 shows that India leads with 35m bales, followed by China (28m bales), the U.S. (21m bales), Brazil (9m bales) and Australia (5m bales). All of them are GM cotton countries, contributing to more than 90% of global cotton production. GM cotton was introduced in Brazil in 2006-07.

India also produces hybrid cotton seed because of the availability of labour to carry out the hand pollination at reasonable cost; this is not available in the U.S., Brazil, Australia and China. Hybrid cotton has delivered not only higher yields but also provided resistance to some pests and diseases.

The article also claims that Indian farmers need to buy seeds repeatedly. The fact is that not just biotech cotton, but all hybrid seeds lose their benefits if replanted, creating reduced and erratic yields. New seeds help farmers sustain high yields year on year.

In the case of biotech cotton in India, it is the farmers who adopted the technology wholeheartedly because they saw a solution in it to some of their biggest on-field pest challenges. The choice made by the Indian farmer to plant hybrid cotton seeds on over 90% of cotton acreage, and see increased cotton production is testament to the value created by better seeds, technologies and farming practices when compared with the alternative of low tech seed and insecticide sprays.

Bt cotton was released in varieties by some public institutions but it did not get much traction. There is always a debate about the use of hybrids versus varieties in any crop. The writer appears to be giving too much power to the seed industry in terms of influencing the farmer to prefer hybrids over varieties. This has not happened in the case of rice, mustard, many oilseeds, and pulses in which the farmers grow varietal crops in 90-100% of the area. The lesson is that the farmer adopts technologies which are beneficial to him and does not go by the recommendations of the industry or any other persons.

Seeds with biotechnologies have helped conserve biodiversity: with higher production from the same area, the expansion of agricultural land into forest areas has been slowed.

A one-sided depiction not only harms agriculture and the industry but also spreads misconceptions about biotechnology.

Ram Kaundinya is Director General, Federation of Seed Industry of India (FSII), New Delhi

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Is Oncolytics Biotech, Inc. (ONCY) a Winner or a Loser in the Biotechnology Industry – InvestorsObserver

January 30th, 2020 3:45 pm

Oncolytics Biotech, Inc. (ONCY) is near the top in its industry group according to InvestorsObserver. ONCY gets an overall rating of 78. That means it scores higher than 78 percent of stocks. Oncolytics Biotech, Inc. gets a 94 rank in the Biotechnology industry. Biotechnology is number 55 out of 148 industries.

Click Here to get the full Stock Score Report on Oncolytics Biotech, Inc. (ONCY) Stock.

Finding the best stocks can be tricky. It isnt easy to compare companies across industries. Even companies that have relatively similar businesses can be tricky to compare sometimes. InvestorsObservers tools allow a top-down approach that lets you pick a metric, find the top sector and industry and then find the top stocks in that sector.

These scores are not only easy to understand, but it is easy to compare stocks to each other. You can find the best stock in an industry, or look for the sector that has the highest average score. The overall score is a combination of technical and fundamental factors that serves as a good starting point when analyzing a stock. Traders and investors with different goals may have different goals and will want to consider other factors than just the headline number before making any investment decisions.

Oncolytics Biotech, Inc. (ONCY) stock is trading at $3.11 as of 9:57 AM on Monday, Jan 27, a loss of -$0.16, or -5.05% from the previous closing price of $3.27. The stock has traded between $3.00 and $3.35 so far today. Volume today is below average. So far 349,770 shares have traded compared to average volume of 2,557,054 shares.

To see InvestorsObserver's Sentiment Score for Oncolytics Biotech, Inc. click here.

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Vir Biotechnology, Inc. (VIR) distance from 20-day Simple moving Average is 47.94% : What to Expect? – The InvestChronicle

January 30th, 2020 3:45 pm

Vir Biotechnology, Inc. (VIR) is priced at $22.47 after the most recent trading session. At the very opening of the session, the stock price was $20.63 and reached a high price of $26.29, prior to closing the session it reached the value of $21.31. The stock touched a low price of $20.21.

Price records that include history of low and high prices in the period of 52 weeks can tell a lot about the stocks existing status and the future performance. Presently, Vir Biotechnology, Inc. shares are logging -18.22% during the 52-week period from high price, and 92.87% higher than the lowest price point for the same timeframe. The stocks price range for the 52-week period managed to maintain the performance between $11.65 and $27.48.

The companys shares, operating in the sector of healthcare managed to top a trading volume set approximately around 2.14 million for the day, which was evidently higher, when compared to the average daily volumes of the shares.

When it comes to the year-to-date metrics, the Vir Biotechnology, Inc. (VIR) recorded performance in the market was 78.69%, having the revenues showcasing 48.32% on a quarterly basis in comparison with the same period year before. At the time of this writing, the total market value of the company is set at 2.19B, as it employees total of 217 workers.

Raw Stochastic average of Vir Biotechnology, Inc. in the period of last 50 days is set at 68.37%. The result represents improvement in oppose to Raw Stochastic average for the period of the last 20 days, recording 67.66%. In the last 20 days, the companys Stochastic %K was 70.12% and its Stochastic %D was recorded 78.51%.

Now, considering the stocks previous presentation, multiple moving trends are noted. Year-to-date Price performance of the companys stock appears to be pessimistic, given the fact the metric is recording 78.69%. The shares increased approximately by 1.27% in the 7-day charts and went down by 35.85% in the period of the last 30 days. Common stock shares were driven by 48.32% during last recorded quarter.

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Vir Biotechnology, Inc. (VIR) distance from 20-day Simple moving Average is 47.94% : What to Expect? - The InvestChronicle

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GIOSTAR Announces Medical Breakthrough in Biotechnology and Lifesciences To Manufacture Abundant, Safe Red Blood Cells From Stem Cells – Benzinga

January 30th, 2020 3:45 pm

GIOSTAR/HEAMGEN has developed and secured patented technology to manufacture lifesaving mature red blood cells from stem cells. The red blood cells are made utilizing a bioreactor that permits the production of mature red blood cells, under strictly controlled conditions, for transfusion therapy and replaces the need for a human blood donor. GIOSTAR/HEAMGEN mature red blood cells are safe and not compromised by inadequate pathogen detection and inactivation of diseases such as hepatitis C, HIV, hepatitis B and syphilis. The red blood cells are O-Negative (Universal Donor) to eliminate incompatibility and allosensitization reactions.

ATLANTA (PRWEB) January 29, 2020

GIOSTAR/HEAMGEN has developed and secured patented technology to manufacture lifesaving mature red blood cells from stem cells. The red blood cells are made utilizing a bioreactor that permits the production of mature red blood cells, under strictly controlled conditions, for transfusion therapy and replaces the need for a human blood donor. GIOSTAR/HEAMGEN mature red blood cells are safe and not compromised by inadequate pathogen detection and inactivation of diseases such as hepatitis C, HIV, hepatitis B and syphilis. The red blood cells are O-Negative (Universal Donor) to eliminate incompatibility and allosensitization reactions. Trauma situations often do not allow for adequate blood typing due to time restrictions, so the GIOSTAR/HEAMGEN red blood cells address that need effectively.

"There are three main problems for blood transfusions," stated Dr. Anand Srivastava, Founder and Chairman of GIOSTAR. "First we have to match the blood type. Second, there's not enough blood available every single time. And third, when we transfer blood from one person to another person, there is always a chance of the transfer of disease."

Watch a feature interview with Dr. Anand Srivastava on The DM Zone with host Dianemarie Collins.

The World Health Organization (WHO) published the first detailed analysis on the global supply and demand for blood in October 2019 and found that 119 out of 195 countries do NOT have enough blood in their blood banks to meet hospital needs. In those nations, which include every country in central, eastern, and western sub-Saharan Africa, Oceania (not including Australasia), and south Asia are missing roughly 102,359,632 units of blood, according to World Health Organization (WHO) goals. While total blood supply around the world was estimated to be around 272 million units, in 2017, demand reached 303 million units. That means the world was lacking 30 million units of blood, and in the 119 countries with insufficient supply, that shortfall reached 100 million units.

The global market opportunity for GIOSTAR/HEAMGEN technology presents not only a profitable and scalable business opportunity but also a significant social and environmental impact. The global market is estimated to be at least $ 85 Billion/year.

GIOSTAR/HEAMGEN has identified early entry global markets to include Military, Trauma, Asia (replace Hepatitis C contaminated blood products), Africa (AIDS contaminated blood), Newborns, Thalassemia patients, Allosensitized sickle cell disease patients. South Sudan was found to have the lowest supply of blood, at 46 units per 100,000 people. In fact, the country's need for blood was deemed 75 times greater than its supply. In India, which had the largest absolute shortage, there was a shortfall of nearly 41 million units, with demand outstripping supply by over 400 percent. Strategic investments are needed in many low-income and middle-income countries to expand national transfusion services and blood management systems. Oncology is a major user of blood transfusion but if countries don't have the capacity to manage the bulk of oncology, it will limit complex surgery options.

GIOSTAR/HEAMGEN has acquired the exclusive license to the patent for the technique for stem cell proliferation from University of California San Diego (UCSD). The founding team of GIOSTAR/HEAMGEN is comprised of the scientists and clinicians who were involved in creating the Intellectual Property at UCSD and has already achieved PROOF OF CONCEPT - the optimized lab scale proliferation of mature red blood cells - at UCSD as part of their research.

GIOSTAR/HEAMGEN is currently looking for strategic partnerships (Contact Doug@DMProductionsLLC.com) to accelerate the development of donor-independent red blood cells manufacturing capabilities and advance the proof of concept work already done (patented) around the manufacture of safe, universal donor, human red blood cells. GIOSTAR/HEAMGEN will also develop a full automated proprietary bioreactor using robotic technology to produce abundant quantities of red blood cells with a goal for cost-effective commercialization of fresh, human, universal donor Red Blood Cells (RBCs).

ABOUT GIOSTAR

Dr. Anand Srivastava is a Chairman and Cofounder of California based Global Institute of Stem Cell Therapy and Research (GIOSTAR) headquartered in San Diego, California, (U.S.A.). The company was formed with the vision to provide stem cell based therapy to aid those suffering from degenerative or genetic diseases around the world such as Parkinson's, Alzheimer's, Autism, Diabetes, Heart Disease, Stroke, Spinal Cord Injuries, Paralysis, Blood Related Diseases, Cancer and Burns. GIOSTAR is a leader in developing most advance stem cell based technology, supported by leading scientists with the pioneering publications in the area of stem cell biology. Company's primary focus is to discover and develop a cure for human diseases with the state of the art unique stem cell based therapies and products. The Regenerative Medicine provides promise for treatments of diseases previously regarded as incurable.

GIOSTAR is world's leading Stem cell research company involved with stem cell research work for over a decade. It is headed by Dr Anand Srivastava, who is a pioneer and a world-renowned authority in the field of Stem Cell Biology, Cancer and Gene therapy. Several governments and organizations including USA, India, China, Turkey, Kuwait, Thailand, Philippines, Bahamas, Saudi Arabia and many others seek his advice and guidance on drafting their strategic and national policy formulations and program directions in the area of stem cell research, development and its regulations. Under his creative leadership, a group of esteemed scientists and clinicians have developed and established Stem Cell Therapy for various types of autoimmune diseases and blood disorders, which are being offered to patients in USA and soon it will be offered on a regular clinical basis to the people around the globe.

For the original version on PRWeb visit: https://www.prweb.com/releases/giostar_announces_medical_breakthrough_in_biotechnology_and_lifesciences_to_manufacture_abundant_safe_red_blood_cells_from_stem_cells/prweb16854975.htm

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Changed landscape of biotech sector, fiercely committed to India, says Department of Biotechnology secretary Dr Renu Swarup – The Indian Express

January 30th, 2020 3:45 pm

Written by Anuradha Mascarenhas | Pune | Published: January 29, 2020 5:20:12 am Dr M K Bhan

Hailing him as the strong pillar on which the Department of Biotechnology (DBT) stood today, secretary Dr Renu Swarup said noted paediatrician and former DBT secretary Dr M K Bhan, who died on January 26, changed the landscape of the biotechnology sector and was among those scientists who had been able to fulfil the dream of introducing a rotavirus vaccine, now part of the universal immunisation programme. Swarup worked closely with Dr Bhan for over a decade.

Dr Bhan pioneered the first indigenously developed rotavirus vaccine and the highest tribute to him would be to take forward his legacy, Dr Swarup said, adding that he was a visionary and his death had left a void that will never get filled.

The rotavirus vaccine was developed from scratch and the government introduced it in the public health programme from 2016.

Generous and passionate about building up young people, Dr Bhan was fiercely committed to our country and its science, but gentle in his approach to individuals and rigorous in his approach to institutions. Dr Bhan was unique and special, Prof Gagandeep Kang, executive director of Translational Health Science and Technology Institute, Faridabad, told The Indian Express.

Prof Chittaranjan Yajnik, noted diabetologist and director of the diabetes unit at KEM hospital in Pune said his death was a massive loss to Indian science. His legacy will guide us in solving problems, said Prof Yajnik, who was his close friend.

Prof Yajnik added that he was a visionary scientist who handled science, its translation and management seamlessly. Dr Bhan was instrumental in promoting new biotechnology institutes in different parts of the country and involving scientists of multiple specialties to interact with each other under the umbrella of biotechnology.

We are now observing the fruits of this approach. He was instrumental in setting up the vaccine field trial unit at KEM hospital, which has contributed significantly to rotavirus research and related studies, Prof Yajnik said.

Dr Bhan was my mentor; as a young trainee gastroenterologist visiting AIIMS, he inspired us to inculcate a systematic approach to clinical problems and as a teacher of epidemiology he shared his belief that public health research was the need of the hour, said Dr Ashish Bawdekar, consultant paediatric gastroenterologist at KEM hospital.

Dr Bawdekar further talked of his close involvement with Dr Bhan, who was the DBT secretary during the development of the Indian rotavirus vaccine.

He was extremely nationalistic and felt Indian scientists were as good as others in the world and would not accept any interference from foreign funders. It was unbelievable what he could foresee and then, more importantly, do something about it, he said.

Dr Sanjay Juvekar, head of the Vadu Rural Health Programme said their team will cherish the experience shared with Dr Bhan on the rotavirus vaccine research,

He was in favour of translational research that could yield results with immediate benefits to the public. Apart from his immense contribution towards the indigenous rotavirus vaccine, he was also instrumental in the identification of zinc deficiency as the cause behind several abnormalities observed in children, said Dr Manoj Kumar Bhat, director of National Centre for Cell Science (NCCS).

Senior scientist at NCCS, Dr Yogesh Shouche, said during his tenure, Dr Bhan came up with several new initiatives not only in health but also in other sectors like agriculture and food processing.

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Europe’s guardian of stem cells and hopes, real and unrealistic – INQUIRER.net

January 30th, 2020 3:44 pm

Submerged in liquid nitrogen vapor at a temperature of minus 175 degrees Celsius, hundreds of thousands of stem cells from all over Europe bide their time in large steel barrels on the outskirts of Warsaw.

Present in blood drawn from the umbilical cord of a newborn baby, stem cells can help cure serious blood-related illnesses like leukemias and lymphomas, as well as genetic conditions and immune system deficits.

Polish umbilical cord blood bank PBKM/FamiCord became the industrys leader in Europe after Swiss firm Cryo-Save went bankrupt early last year.

It is also the fifth largest in the world, according to its management, after two companies in the United States, a Chinese firm and one based in Singapore.

Since the first cord blood transplant was performed in France in 1988, the sector has significantly progressed, fuelling hopes.

Health insurance

Mum-of-two Teresa Przeborowska has firsthand experience.

At five years old, her son Michal was diagnosed with lymphoblastic leukemia and needed a bone marrow transplant, the entrepreneur from northern Poland said.

The most compatible donor was his younger sister, Magdalena.

When she was born, her parents had a bag of her cord blood stored at PBKM.

More than three years later, doctors injected his sisters stem cells into Michals bloodstream.

It was not quite enough for Michals needs but nicely supplemented harvested bone marrow.

As a result, Michal, who is nine, is now flourishing, both intellectually and physically, his mum told AFP.

A cord blood transplant has become an alternative to a bone marrow transplant when there is no donor available, with a lower risk of complications.

Stem cells taken from umbilical cord blood are like those taken from bone marrow, capable of producing all blood cells: red cells, platelets and immune system cells.

When used, stem cells are first concentrated, then injected into the patient. Once transfused, they produce new cells of every kind.

At the PBKM laboratory, each container holds up to 10,000 blood bags Safe and secure, they wait to be used in the future, its head, Krzysztof Machaj, said.

The bank holds around 440,000 samples, not including those from Cryo-Save, he said.

If the need arises, the blood will be ready to use without the whole process of looking for a compatible donor and running blood tests, the biologist told AFP.

For families who have paid an initial nearly 600 euros (around P34,000) and then an annual 120 euros (around P7,000) to have the blood taken from their newborns umbilical cords preserved for around 20 years, it is a kind of health insurance promising faster and more effective treatment if illness strikes.

But researchers also warn against unrealistic expectations.

Beauty products

Hematologist Wieslaw Jedrzejczak, a bone marrow pioneer in Poland, describes promoters of the treatment as sellers of hope, who make promises that are either impossible to realize in the near future or downright impossible to realize at all for biological reasons.

He compares them to makers of beauty products who swear their cream will rejuvenate the client by 20 years.

Various research is being done on the possibility of using the stem cells to treat other diseases, notably nervous disorders. But the EuroStemCell scientist network warns that the research is not yet conclusive.

There is a list of almost 80 diseases for which stem cells could prove beneficial, U.S. hematologist Roger Mrowiec, who heads the clinical laboratory of the cord blood program Vitalant in New Jersey, told AFP.

But given the present state of medicine, they are effective only for around a dozen of them, like leukemia or cerebral palsy, he said.

Its not true, as its written sometimes, that we can already use them to fight Parkinsons disease or Alzheimers disease or diabetes.

EuroStemCell also cautions against private blood banks that advertise services to parents suggesting they should pay to freeze their childs cord blood in case its needed later in life.

Studies show it is highly unlikely that the cord blood will ever be used for their child, the network said.

It also pointed out that there could be a risk of the childs cells not being useable anyway without reintroducing the same illness.

Some countries, such as Belgium and France, are cautious and ban the storage of cord blood for private purposes. Most E.U. countries however permit it while imposing strict controls.

Rapid growth

In the early 2000s, Swiss company Cryo-Save enjoyed rapid growth.

Greeks, Hungarians, Italians, Spaniards and Swiss stored blood from their newborns with the company for 20 years on payment of 2,500 euros (around P140,000) upfront.

When the firm was forced to close in early 2019, clients were left wondering where their stem cells would end up.

Under a kind of back-up agreement, the samples of some 250,000 European families were transferred for storage at PBKM.

The Polish firm, founded in 2002 with 2 million zlotys (around P26 million), has also grown quickly.

Present under the FamiCord brand in several countries, PBKM has some 35% of the European market, excluding Cryo-Save assets.

Over the last 15 months, outside investors have contributed 63 million euros to the firm, PBKMs chief executive Jakub Baran told AFP.

But the company has not escaped controversy: the Polityka weekly recently published a critical investigative report on several private clinics that offer what was described as expensive treatment involving stem cells held by PBKM.IB/JB

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Orchard Therapeutics Announces FDA Granted Orphan Drug Designation for OTL-102 for the Treatment of X-linked Chronic Granulomatous Disease (X-CGD) -…

January 30th, 2020 3:44 pm

Early Clinical Data Support ex vivo Hematopoietic Stem Cell Gene Therapy as a Potentially Promising Treatment Option for X-CGD

BOSTON and LONDON, Jan. 29, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics (ORTX), a global gene therapy leader, today announced that it has received orphan drug designation from the U.S. Food and Drug Administration (FDA) for OTL-102, the companys ex vivo autologous hematopoietic stem cell (HSC) gene therapy being investigated for the treatment of X-linked chronic granulomatous disease (X-CGD). The FDA may grant orphan designation to drugs and biologics intended to treat a rare disease or condition affecting fewer than 200,000 persons in the U.S.

We are pleased to have received this orphan drug designation from the FDA, which recognizes the potential of OTL-102 to address a rare population of patients with X-CGD, a life-threatening disease with a critical unmet need, said Anne Dupraz-Poiseau, Ph.D., chief regulatory officer at Orchard. We are encouraged by the clinical data published to date and are eager to advance OTL-102 development as quickly as possible for patients with X-CGD.

Orphan designation qualifies a company for certain benefits, including financial incentives to support clinical development and the potential for seven years of market exclusivity in the U.S. upon regulatory approval.

Early academic clinical trial data for OTL-102 that was recently published in Nature Medicine demonstrates that ex vivo autologous HSC gene therapy may be a promising approach for the treatment of X-CGD. The letter, which wasled by researchers at the University of California, Los Angeles (UCLA)including Donald B. Kohn, M.D., one of the study's lead investigators and professor of microbiology, immunology and molecular genetics at UCLA and Great Ormond Street Hospital (UK), provides an analysis of safety and efficacy outcomes in nine severely affected patients with X-CGD. At 12 months post-treatment, six of seven surviving patients, all of whom were adults or late adolescents, exceeded the minimum threshold hypothesized in published literature to demonstrate potential clinical benefit, defined as 10% functioning, oxidase-positive neutrophils in circulation and have discontinued preventive antibiotics.1

As previously reported, two pediatric patients died within three months of treatment from complications deemed by the investigators and independent data and safety monitoring board to be related to pre-existing comorbidities due to advanced disease progression and unrelated to OTL-102. Investigators are planning to enroll additional pediatric patients in 2020 to assess outcomes in this patient population. In addition, there is work underway to improve the efficiency of the drug product manufacturing process prior to initiating a registrational study.

Patients with X-CGD experience significantly reduced quality and length of life, and currently must take daily medications that do not eliminate the risk of fatal infections, said Adrian Thrasher, Ph.D., M.D., one of the studys lead investigators and professor of pediatric immunology and Wellcome Trust Principal Research Fellow at UCL Great Ormond Street Institute of Child Health in London. These data demonstrate that OTL-102 has the potential to become a transformative new treatment option for patients with X-CGD with the evaluation of longer follow up and more patients.

About X-CGDX-linked chronic granulomatous disease (X-CGD) is a rare, life-threatening, inherited disease of the immune system caused by mutations in the cytochrome B-245 beta chain (CYBB) gene encoding the gp91phox subunit of phagocytic NADPH oxidase. Because of this genetic defect, phagocytes, or white blood cells, of X-CGD patients are unable to kill bacteria and fungi, leading to chronic, severe infections. The main clinical manifestations of X-CGD are pyoderma, a type of skin infection; pneumonia; colitis; lymphadenitis, an infection of the lymph nodes; brain, lung and liver abscesses; and osteomyelitis, an infection of the bone. Patients with X-CGD typically start to develop infections in the first decade of life, and an estimated 40 percent of patients die by the age of 35.2 The incidence of X-CGD is currently estimated at between 1 in 100,000 and 1 in 400,000 male births.

Story continues

About OTL-102OTL-102 is an ex vivo autologous hematopoietic stem cell gene therapy being studied for the treatment of X-CGD. The studies are supported by multiple institutions including the California Institute of Regenerative Medicine, the Gene Therapy Resource Program from the National Heart, Lung, and Blood Institute, the National Institute of Allergy and Infectious Diseases Intramural Program, the Wellcome Trust and the National Institute for Health Research Biomedical Research Centres at Great Ormond Street Hospital for Children NHS Foundation Trust, University College London Hospitals NHS Foundation Trust and University College London. Preclinical and clinical development of OTL-102 had originally been initiated by Genethon (Evry, France) and funded by an EU framework 7 funded consortium, NET4CGD, before being licensed to Orchard.

About OrchardOrchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically-modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. The company has one of the deepest gene therapy product candidate pipelines in the industry and is advancing seven clinical-stage programs across multiple therapeutic areas, including inherited neurometabolic disorders, primary immune deficiencies and blood disorders, where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.

Orchard has its global headquarters in London and U.S. headquarters in Boston. For more information, please visit http://www.orchard-tx.com, and follow us on Twitter and LinkedIn.

Forward-Looking StatementsThis press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements may be identified by words such as anticipates, believes, expects, plans, intends, projects, and future or similar expressions that are intended to identify forward-looking statements. Forward-looking statements include express or implied statements relating to, among other things, the therapeutic potential of Orchards product candidates, including the product candidate or candidates referred to in this release, Orchards expectations regarding the timing of regulatory submissions for approval of its product candidates, including the product candidate or candidates referred to in this release, the timing of interactions with regulators and regulatory submissions related to ongoing and new clinical trials for its product candidates, the timing of announcement of clinical data for its product candidates and the likelihood that such data will be positive and support further clinical development and regulatory approval of these product candidates, and the likelihood of approval of such product candidates by the applicable regulatory authorities. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, the risks and uncertainties include, without limitation: the risk that any one or more of Orchards product candidates, including the product candidate or candidates referred to in this release, will not be successfully developed or commercialized, the risk of cessation or delay of any of Orchards ongoing or planned clinical trials, the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving Orchards product candidates,the delay of any of Orchards regulatory submissions, the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchards product candidates, the receipt of restricted marketing approvals, and the risk of delays in Orchards ability to commercialize its product candidates, if approved. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards annual report on Form 20-F for the year ended December 31, 2018, as filed with the U.S. Securities and Exchange Commission (SEC) on March 22, 2019, as well as subsequent filings and reports filed with the SEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

References1Kang et al. Blood. 2010;115(4):783-912van den Berget al. PLoS One. 2009;4(4):e5234

Contacts

InvestorsRenee LeckDirector, Investor Relations+1 862-242-0764Renee.Leck@orchard-tx.com

MediaMolly CameronManager, Corporate Communications+1 978-339-3378media@orchard-tx.com

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Dad launches appeal in memory of Eimear – Belfast Media Group

January 30th, 2020 3:44 pm

By Michael Jackson

THE father of a young West Belfast cancer battler, who passed away last year, has helped launch the Donuts for Donors appeal, the Norths first ever Stem Cell Donation Awareness Day.Sen Smyth, whose daughter Eimear Gooderman (ne Smyth) died on the 27th of June 2019, was joined by the Lord Mayor Danny Baker for the appeal launch a City Hall on Tuesday.In September 2016, Eimear was diagnosed with stage two Hodgkins Lymphoma, a type of blood cancer.After finding a stem cell donor match, Eimear underwent a transplant in November 2018, but sadly passed away following complications.Throughout her illness, the Coolnasilla woman fought to raise awareness of stem cell donation alongside her family, who established the Eimears Wish campaign to continue her work.Speaking to the Andersonstown News, Sen explained the idea behind the Donuts for Donors appeal, which will take place next Friday.The most important thing here is getting the message out that we need people to become stem cell donor and organ donors, he said.We want our politicians to fall into line with England and Wales and bring in the soft opt out where everybody is a donor, and if you dont want to be a donor then you need to opt out.This is our first ever Northern Ireland-wide Stem Cell Donation awareness day and Eimear loved donuts and coffee, so we married the two together.We have raised three or four thousand pounds for charity since Eimear died maybe more and we just dont ask people for money. We try to organize events to give people a reason to put their hands in their pockets, so we thought why dont we do Donuts for Donors get them to buy a donut, get them to have a coffee morning in their workplace.The money is going to fantastic charities; Action Cancer, Friends of the Cancer Centre and the Anthony Nolan Trust. That way were hoping to raise a few pound.Our percentage of donors is very low, so its also about getting people talking about and become aware of it.To mark the occasion on Friday, January 31, City Hall will also be lit up pink, which was Eimears favourite colour.Local businesses and schools will also be involved including West Belfasts All Saints College and St Louises Comprehensive College.Speaking ahead the event, Councillor Baker encouraged everyone to get the kettle on and pick up a donut to help raise much-needed funds and awareness of stem cell donation.This is a very important initiative which is helping to shine a spotlight on the important topic of stem cell donation and organ donation generally, he said.The team at Eimears Wish have worked tirelessly to keep the spirit of Eimear alive and continue her great work by making stem cell donation an important topic of conversation.On Friday 31 January I will be hosting a special coffee morning in the Parlour for people and families affected by stem cell transplants and Im calling on everyone across the city to follow suit and get involved for this great cause.

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Dodge Elementary rallies around beloved therapy dog – Amherst Bee

January 30th, 2020 3:44 pm

Students at Dodge Elementary School hold up colored portraits of Yeti, the schools therapy dog, in the classroom.

Dodge Elementary students have been missing one of their favorite faculty members; Yeti the therapy dog has been absent as he heals from hip dysplasia complications.

Dan Steck, head custodian at Dodge Elementary, rescued Yeti from a puppy mill near Lancaster, Pennsylvania, on March 19, 2016. Little did he know then that soon, Yeti would become a certified therapy dog, complete with his own office at Dodge.

Steck originally rescued Yeti in the hope that a sibling would calm down his other rambunctious malamute, Luna. While the adoption didnt quite have the desired effect, Steck soon realized that Yeti was special he was kind, gentle and always happy, a perfect candidate to become a therapy dog.

Since his certification through the SPCAs Paws for Love program, Yeti has worked at Dodge, a friendly face for struggling children or a non-judgmental listener for children practicing their reading. Steck said that teachers and students make requests to have Yeti in their classrooms left and right.

They adore Yeti, said Dodge principal Charlie Smilinich. Its cool to see how a dog can impact students. Its very unique. Hes been here for two years and has built a relationship with everyone. Hes a Dodger. The dog has his own office.

Yeti is what his owners call a tripaw, meaning that one of his legs was amputated due to a neglected infection he sustained under the care of his previous owners. As a result of walking on only three legs, the 4-year-old Alaskan malamute struggles with hip dysplasia.

Originally, Yeti was prescribed hydrotherapy, though he struggled to keep up with the treatment and would come home exhausted. Now, veterinarians are considering acupuncture, stem cell treatment or even a total hip replacement.

Hopefully, hell live a long life, but hes got some real challenges already, said Steck. At age 4, hes nowhere near as playful as he was, and he sleeps a lot. Eventually, he may even need a wheelchair.

Stecks family has dutifully supported Yeti through all of his medical trials, but the bills are racking up quickly. A wheelchair alone would cost them between $1,200 and $1,500.

To help with the costs, the Stecks have a GoFundMe page where they provide medical updates and receive encouraging messages from the elementary students.

To support their beloved therapy dog, Dodge students and the surrounding community have contributed $3,845 thus far, with a goal of $10,000. All of the money raised goes directly into an account at Amherst Small Animal Hospital.

To support the GoFundMe campaign, visit http://www.gofundme.com and search for Throw Kindness Around Like Confetti For Yeti.

To learn more about Yeti and his work at Dodge, search for Yeti Steck on Facebook.

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Five Biotechs Poised to Advance Their Rare Disease Programs in 2020 – BioBuzz

January 30th, 2020 3:44 pm

February is Rare Disease Awareness Month, a month-long celebration focused on the suffering and triumphs of patients and their families stricken by rare diseases culminating on February 29th International Rare Disease Day. The rapidly developing cell and gene therapy fields in recent years have led to a rare disease R&D boom of sorts, with an estimated 800 rare disease-focused clinical development projects currently in process. The biotech industry sees a new window for improving rare disease patients lives and potentially curing rare diseases that impact one in ten American and an estimated 400 million people globally.

The Biohealth Capital Region (BHCR) is at the leading edge of the rare disease market with companies like American Gene Technologies, REGENXBIO, Cerecor, Asklepion Pharmaceuticals, Lediant, and others advancing their pipelines towards rare disease treatments and cures. The regions robust rare disease sector also includes a host of companies that support rare disease research and development like Jeeva Informatics, GeneDx, Aperiomics and HemoShere among others. In short, the portfolio of rare disease companies and growing support ecosystems make the BHCR a global hub for rare disease R&D.

With Maryland already housing the largest number of Centers of Excellence for the rare disease community, the world travels to us. Roughly speaking, 50% of rare disease patients are children who will never see their tenth birthday and we need this number to change. Currently, Maryland is on the radar to be the next top biotech state and as a mother of two rare disease patients, there is zero time to wait, stated Lydia Seiders, an independent global strategic advisor for the rare disease community and volunteer state ambassador for the National Organization for Rare Disorders (NORD) Maryland Rare Action Network.

Every biotech investing in and pursuing more effective rare disease treatments and potential cures understands Seiders urgency. Lets take a look at some of the leading companies in the BHCR and what we might expect in 2020 from the rare disease programs in their pipelines.

Here is a snapshot of five leading companies and their rare disease pipelines.

American Gene Technologies is a cell and gene therapy company that utilizes a proprietary lentiviral platform to develop potential treatments for liver cancer, Phenylketonuria (PKU), HIV and inherited genetic disorders. AGTs novel lentiviral platform has enabled the company to develop, test and bank thousands of lentiviral vectors, which can then be matched to develop treatments for new target diseases, including orphan/rare diseases like PKU as well as diseases with larger patient populations.

PKU is an inherited, monogenetic disease that causes excess accumulation of the amino acid phenylalanine (Phe), which can reach toxic levels in the blood without strict dietary control. Children born with PKU (PKU occurs in approximately 1 in every 13,500 births) can suffer brain and organ defects if not placed on a special diet and adults can suffer neuropsychiatric impairments including poor behavioral restraint and limited executive function with a high risk of self-injury.

AGT is committed to curing PKU via its proprietary lentiviral platform. The company received FDA Orphan Drug Designation in 2018 and is progressing its PKU therapy toward the clinic.

REGENXBIO is a leading clinical-stage biotechnology company developing gene therapies for retinal, neurodegenerative and metabolic diseases. The companys proprietary NAV Platform, which was invented to discover next-generation adeno-associated viral (AAV) vectors to propel the discovery of new therapies across a wide range of disease areas, including rare/orphan diseases. REGENXBIO has exclusive rights to more than 100 novel AAV vectors and more than 100 patents and patent applications for AAV vectors worldwide.

REGENXBIO currently has 13 clinical-stage products and has 20 partnered programs. One of their out-licensed programs delivered an important milestone for the company, and for the rare disease community. The FDA approval of Avexis ZOLGENSMA, which was developed from REGENXBIOs NAV Technology Platform, became the first cure for spinal muscular atrophy (SMA) and a monumental triumph of the use of gene therapy for the rare disease field. SMA is a fatal childhood neuromuscular disease caused by a mutation in a single gene.

The companys pipeline includes several rare disease candidates.

RGX-121 is a product candidate for Hunter Syndrome (Mucopolysaccharidosis Type II), which is a rare, X-linked recessive disease that causes developmental delay and developmental regression in children. In December 2019, the company announced that it had successfully advanced the clinical safety and efficacy testing into the second cohort of patients at a higher dosage level. Interim data on the second cohort should be released by approximately mid-2020. The first cohort of dosed patients resulted in positive safety and efficacy outcomes, including a meaningful and sustained reduction in heparan sulfate suggesting that the gene therapy can potentially restore intracellular activity of the I2S enzyme, as well as the early signs of neurocognitive stability that have been observed, stated Steve Pakola, M.D., Chief Medical Officer of REGENXBIO.

RGX 501 is a therapy being developed for the Treatment of Homozygous Familial Hypercholesterolemia (HoFH), which impacts approximately 11,000 patients globally.

REGENXBIO previously announced the completed dosing of an expanded Cohort 2 in Phase I/II trial of RGX-501 including steroid prophylaxis. Per protocol, patients received at least a 13-week steroid treatment. No serious adverse effects or significant elevations in liver enzyme levels were reported in the expanded Cohort 2. REGENXBIO plans to assess low-density lipoprotein (LDL-C) levels in the expanded Cohort 2 after all patients have completed their steroid treatment and expects to provide interim data in the first half of 2020.

REGENEXBIO is also working to advance RGX-181, which received FDA Rare Pediatric Disease and Orphan Drug Designations as a treatment candidate for late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, one of the most common forms of Batten disease. The company anticipates submitting an investigational new drug (IND) application to the U.S. Food and Drug Administration (FDA) in the second half of 2020 for RGX-181 to enable initiation of a first-in-human clinical trial for children with CLN2 disease.

RGX-111 is REGENEXBIOs potential treatment for another rare disease called Mucopolysaccharidosis Type I (MPS I), a rare, recessive genetic disease that can cause hydrocephalus, spinal cord compression and cognitive impairment. Recruitment, screening, and additional site activations are ongoing in Phase I clinical trials evaluating RGX-111 for the treatment of MPS I. RGX-111 has received orphan drug products, rare pediatric disease and Fast Track designation from the FDA. A program update will be available in the second half of 2020.

REGENXBIOs pipeline is representative of the balanced approach many bioscience companies strive for with a focus on rare disease and potential blockbuster treatments (WET AMD).

Cerecor, Inc. is a publicly-traded biotechnology company focused on developing innovative therapies for Orphan Diseases and ultra-rare pediatric diseases. Founded in 2011, the company has brought numerous prescription medicines, prescription medical devices, and dietary supplement products to the market. Cerecors pipeline includes a host of rare disease treatments in the early stages of development.

Its leading pediatric treatment is CERC-801, an ultra-pure oral formulation of D-galactose currently in development for the treatment of phosphoglucomutase 1 (PGM1) deficiency, also known as PGM1-CDG, which is an inherited genetic disorder with wide-ranging symptoms. This potential treatment is in Phase I clinical trials and is a pediatric program with an expedited 505(b)(2) pathway designation.

Other pediatric rare diseases being targeted by Cerecor include the rare, congenital disorder MPI-CDG, which is in Phase I pediatric trials with an expedited 505(b)(2) pathway designation;Leukocyte Adhesion Deficiency Type II (LADII), also known as SLC35C1-CDG, which is in its IND-enabling phase; and the ultra-rare mitochondrial DNA depletion syndrome (MDS), also known as Deoxyguanosine Kinase (DGUOK) deficiency, which is also in the IND-enabling phase.

Asklepion Pharmaceuticals is a biopharmaceutical company that is exclusively focused on rare disease therapy development, specifically for rare pediatric diseases without viable treatments. The company has shifted its focus exclusively to pediatric rare disease in recent years. Asklepion was founded in 2006 in an effort to commercialize a 15-year academic program focused on developing cholic acid for inborn errors of bile acid metabolism, which is a rare genetic disorder.

Its leading therapy candidate is for acute lung injury induced by cardiopulmonary bypass surgery (CBP), which recently completed Phase III clinical trial enrollment in July 2019. Cardiopulmonary bypass surgery can damage lung tissue and blood vessels, which can lead to significant postoperative problems. Asklepions treatment is unique in that it is preventative rather than remedial. By intravenously administering the protein building block L-Citrulline pre-surgery, Asklepion believes that postoperative complications caused by CBP. The treatment received FDA Orphan Drug Designation in 2017. The company is also starting a clinical trial for Sickle cell disease (acute setting) in Q2/Q3 of 2020.

For more than three decades, iterations of the company now known as Leadiant Biosciences has displayed an unwavering commitment to treating and helping ADA-SCID patients and their families. Bubble Boy Diseaseor adenosine deaminase deficiency-severe combined immunodeficiency (ADA-SCID)is an ultra-rare, inherited autosomal metabolic disorder that severely compromises the immune system and can be fatal if untreated.

In October 2018, Leadiant Biosciences, formerly known as Sigma-Tau Pharmaceuticals, received FDA approval for Revcovi, which delivers improved treatment for patients living with ADA-SCID. Approximately 1 in 200,000 to 1,000,000 newborns are impacted worldwide, representing about 15% of all SCID cases (Source: NIH). As an ultra-rare disease, the patient population is minuscule: Leadiant currently treats 35 ADA-SCID patients in the U.S. and seven in Canada.

Headquartered in Gaithersburg, Maryland, Leadiants roots run deep in the treatment of rare and ultra-rare diseases. Leadiant Biosciences, Inc. is a subsidiary of Leadiant S.p.A., which traces its roots back to Sigma-Tau, a family-owned pharmaceutical company founded in Italy by Emilio and Dr. Claudio Cavazza in 1957. Sigma-Tau Pharmaceuticals was a rare disease treatment pioneer, receiving the fourth orphan drug designation ever in 1984.

Today, Leadiants sole focus is on researching, developing and distributing novel treatments for patients afflicted with some of the rarest diseases in the world. Revcovis recent FDA approval for the treatment of ADA-SCID gives Leadiant eight rare disease products on the North American (U.S. and Canada) market. With a deep commitment to advancing a diverse pipeline of new drugs and therapeutic candidates, Leadiant has seven clinical-stage programs in their pipeline. The two programs in phase III trials include LB101 for the treatment of Cerebrotendinous Xanthomatosis (CTX), and LB301 for the treatment of GNE Myopathy.

There are several other rare disease-focused BHCR companies ready to emerge. Elixirgen Therapeutics of Baltimore, MD is a biotechnology company seeking cures through stem cell biology. Its pipeline currently has four rare disease therapies in its pipeline, all of which are in the early stages of development. The company recently announced a $4M Series A round. Another Baltimore-based biotech, Abri Science, recently launched in September of 2019. Abri Science is focused on developing treatments for PKU with licensed technology. Since its launch, Abri Science has been awarded a $225,000 Small Business Technology Transfer (STTR) Phase I grant from the US National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK).

The BHCR is rapidly becoming a global hub for rare disease drug development. A host of BHCR companies, including many cell and gene therapy companies, have made rare disease drug development a key component of their pipeline strategies.

BioBuzz will be featuring rare disease stories throughout Rare Disease Awareness month. And well be keeping close tabs on these rare-disease focused companies as we move deeper into 2020.

Check back in for the latest news and developments.

Steve has over 20 years experience in copywriting, developing brand messaging and creating marketing strategies across a wide range of industries, including the biopharmaceutical, senior living, commercial real estate, IT and renewable energy sectors, among others. He is currently the Principal/Owner of StoryCore, a Frederick, Maryland-based content creation and execution consultancy focused on telling the unique stories of Maryland organizations.

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20 Markets to lookout for in 2020 – Expedition 99

January 30th, 2020 3:44 pm

Insight industry exhibits the potential to reveal major business events that impart most relevant insights and holds a strong influence on business communities. There have been notable trends in 2019 as the global insights industry crossed USD 45bn and still continues to raise the bar in the coming years.

Going forward, the market research industry continues to exhibit data insights leading to maximize revenue share for companies that are willing to conduct or outsource market research activities.

Dataintelo A leading market research and consulting company published a list of 20 markets to look for in 2020.

The surgical robots industry is expected to cross USD 25bn in the next five years as per findings by a market research report as there will be a huge demand for automation across the healthcare sector as automation is poised for complex procedures. Moreover, non-invasive surgeries are becoming a common trend for geriatric population with minimal surgical complications. It is also anticipated that surgical robots can help in reducing surgery costs and will gain a high popularity all across the healthcare and medical domain. At present, nanobots are employed for the treatment of arteries and catheter-based robots reveal better operative techniques for cardiac surgeries. North America is the leading market for surgical robots, says the report and it will continue to have a strong influence on the global market.

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The telemedicine industry is expected to cross USD 22bn in the next five years attributing to rise in consultation costs for healthcare and pharmaceutical followed by treatment for chronic diseases. With the advent of technological innovations and availability of healthcare services, telemedicine market is expected to take a high leap in the near future. The telemedicine features in reducing patient visits to hospitals and clinics and simultaneously reducing the burden on hospital infrastructure. For instance, in the U.S. market telemedicine proves effective for cost-controlling in diabetic patients. Various telecare programs have already gained momentum and witness a high adoption. With the growing interest of consumers in mobile health, telemedicine applications are likely to hold a strong influence in the years to come.Buy complete research report on surgical robot: https://dataintelo.com/checkout/?reportId=602&license_type=Single%20User

The defibrillator market is anticipated to witness a rise in revenue share by USD 9.5bn in the near future driven by a huge rise in cases of cardiac arrests. Both, developed and developing nations are now emphasizing on strengthening public infrastructure by offering emergency medical services and also offer a high awareness on cardiac health. Government norms and spending by major vendors is playing a crucial role in building future opportunities. In addition, use of wearable defibrillators is the latest trend witnessed as a healthcare application that augments the chance of survival in case of severe cardiac arrests by alarming the user even before it occurs. China, India, Japan and other markets within Asia Pacific are adopting to a robust healthcare infrastructure resulting in patient safety.

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The halitosis market witnessed a huge revenue share in 2019 accounting for USD 8bn. Surge in cases of halitosis is expected to widen the market scope. In a recent study conducted by dental institute, middle-aged people suffer from bad breath problems as a result of accumulation of oral germs and bad cavity. Alone in the United States, more than 70% population annually spends around USD 15bn on oral care and hygiene products to counter bad breath problems. Consumption of tobacco and alcohol have been major drivers for developing oral care problems within a vast population. It is also estimated that there will be an upsurge in the consumption rate in the next three years. Hence, Asia Pacific witness a significant rise in the treatment for halitosis treatment.

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The cancer stem cell therapeutics market is an emerging one which is expected to bypass USD 150mn in the next five years. Major trends such as rise in awareness and use of advanced genome cell analysis methods have a strong influence the market for cancer stem cell therapeutics. Additionally, Asia Pacific countries like Japan and South Korea are now offering novel opportunities in the line of stem cell therapeutics. Regulatory guidelines and policies are undergoing several revisions for effective development of commercial market. Apart, clinical trials for stem cells validate the potency of stem-cell based products. For instance, there have been several trials undertaken by leading clinical institutes in China such as Xuzhou Medical University and the Affiliated Hospital of Academy of Military Medical Sciences.

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Artificial Intelligence (AI) is all set to reform the agriculture industry and with this the global market for precision farming market is expected to witness a growth of USD 12bn in the next five years. According to market research experts, AI techniques such as farm robots, data mining and use of computational analysis for seasoned crops will be highly witnessed, Agriculture will therefore be a data-driven industry soon. In fact, the use of Internet of things (IoT) and advanced analytics is widely witnessed in several parts of developed countries. Adoption of UAVs, drones, irrigation controllers and steering systems is expected to gain a high momentum by mid 2020 which is expected to accelerate the proliferation of AI in agriculture. With this, sustainability and productive issues in farming can be confronted with adequate technological resources says a market research report.

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Automation continues to induce a high interest in the medical industry and automated dispensing units are gaining a huge popularity. The revenue share is expected to sum at USD 1.8bn by 2021 attributing to the ongoing technological innovations and government norms. Automated dispensers find notable uses resulting to minimize medication errors and improve safety processes of healthcare. Healthcare management systems are adopting automated dispensers for effective drug management. Globally, North America market is anticipated to have a strong influence in the international market with concerns for patient health and safety. On the other hand, Asia Pacific witness a rise in geriatric population with immediate medical attention and crucial developments on the infrastructural front.

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Manufacturing and production sectors are undergoing automation processes and it is likely that automated jobs will be soon a major trend. With the anticipation for the revenue share to cross USD 90bn in the next three years, the demand for high pressure processing equipment will witness huge a significance all over the food and beverage industry. Consumer demand for processed food and growing emphasis on food safety along with adoption of innovation fuels the market growth. Emerging economies such as Latin America and Brazil represent major opportunities for futuristic growth. Chocolate and confectionery processing units represent the largest share in the high pressure processing equipment market. Asia Pacific holds a major accountability thus representing the largest share. Widening scope of urbanization and demand for modern methods of food production are major drivers.

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Eco-friendly initiatives for consumables and stylish trends of bio-based materials in food industry is globally evident. Government norms and policies followed by ban on plastic products in the food industry highlights the shift from traditional to eco-friendly approach. For instance, paper straws have a huge impact on the market attributing to high adoption in the retail sector. Consumer trends such as high awareness, and demand for bio-based reusable materials is likely to be witnessed all across the mature markets. Overall, the reusable straw industry is poised to exhibit a high growth rate in the next three years.

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Indoor fitness trends such as calorie burnout, cardio, open air gymnasium and power yoga are gaining high momentum and therefore the market for home fitness equipment is anticipated to value at USD 15bn in the next three years. Most commonly used equipment are treadmills, stationary bikes and elliptical are in high demand across the global markets. Moreover, the need for maintaining high fitness levels to combat lifestyle diseases is widely witnessed in the urban settings. Professional commitments and time constraints have been fueling the concept of home fitness equipment in the metropolitan cities. Ranging from small to large, fitness equipment for indoors enjoy a wide popularity in urban scenarios.

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The digitalization for broadcast and entertainment sector is expanding and the proliferation of digital channels via internet is most evident. As a result, there is a huge consumer demand for OTT content. Although, the OTT service market is relatively at nascent stages, the market is projected to reach at USD 33bn in the next five years. Dominance of smartphone technology and increased adoption of large screen smartphone is a driving factor across developed nations. Moreover, emergence of hardware compatible devices that support OTT content is resulting in a wide popularity. In addition, developments in the telecommunications sector for video calling and messaging service induces the market position for OTT platforms.

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Food and beverages remains a hot domain for companies to invest in 2020 as confectionery processing equipment segment is expected to show optimal growth valued at USD 5bn in the next three years. There has been a huge popularity of chewing gums, jellies and bakery products in the retail industry. In addition, strong presence of retail companies stimulates the market growth for confectionery processing equipment. Stringent guidelines from USFDA and FAO are shaping up the industrial structure for processing equipment and continue to dominate the business policies. The North American market is a fast-paced market attributing to strong presence of major vendors and players.

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The global market size of plastic to fuel technology was USD 82mn in 2018 and it is expected to reach at USD 2.50bn in the next five years according to a research report. Major trends such as government policies to influence plastic to oil conversion and developing distribution channels is gaining tremendous traction. Growing awareness for plastic consumption and improved disposal methods are major highlights. Moreover, it is also estimated that the plastic to fuel technology market offers lucrative employment opportunities. For instance, in the U.S. plastic based fuel is gaining a high momentum due to low content of sulfur.

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The market size for recycled plastics is expected to bypass USD 67bn in the next five years attributing to the Y-o-Y (year on year) growth across the global market. Polyethylene Terepthalate is a widely used recycled material. Commercially, PET offers lucrative growth opportunities and exhibits high reusable capacity. High density polyethylene holds a maximum share in the global market and expected to grow at a stipulated CAGR. For instance, high density polyethylene is usually employed for margarine tubes, grocery bags, and cleansing bottles. Recycled plastics seem to have a high influence in the consumer appliance industry, therein the market for recycled products witness a strong demand.

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The worldwide construction and real estate business is undergoing rapid innovations, norms and standardized regulations. Demand for quality home is witnessed all across the economies thereby enabling the market to come up with modernized approach in the construction phase. The construction additives is a growing market particularly in the developing economies as the global market is expected to reach at USD 23bn in the near future. Economic reforms and revival post economic crisis in 2008 is much likely to create a balanced market ensuring a high-end growth. In addition, the widening landscape of urbanization within the Asia Pacific market stimulates the ongoing developments.

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The global market share for automotive and vehicle equipment is expected to cross USD 300bn in the next 5-10 years as the year 2020 is the most automotive year. Alarming need for enhanced safety in vehicles and smooth ride are some of the basic factors that demand a vehicle complying to international safety standards. Suspension is a pivotal component in an automotive that is subsequent in enabling a smooth and shock free ride. It also prevents any wear and tear of cargo and protects the passengers from drifting. Use of pneumatic and hydraulic suspension types are some of the modern methods used in designing suspension for a vehicle, says a market report. Increase in rate of inter-state IMEX business with road transport being the crucial mode of transportation propels the demand for heavy commercial vehicles. There have been drastic rise recorded in the past few years which is anticipated to keep rising annually at a higher rate.

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Liquid waste management is a prime concern in the developing economies since marine pollution is relatively high. Therefore, strong initiatives led by NGOs and helping communities have resulted in developing a sustainable system for managing liquid waste and hence the commercial market is gaining a lot of traction. It is also anticipated that the market might gain 4.0% CAGR in the next couple of years thanks to awareness and change in trends for disposal of liquid waste. Industrial liquid waste is associated with potential toxicity posing a serious threat to marine life, on a large scale. Accumulation of hazardous effluents is expected to drive the demand for liquid waste management initiatives in the near future. Apart, stringent norms and government regulations for commercial industries is expected to drive the market development in the forthcoming years.

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Semi-agriculture is relatively a growing domain wherein innovation in farming are been tried and tested. According to a research study, the global market share for humic acid water soluble fertilizers is poised to bypass USD 130bn in the near future. Notable growth factors such as enormous demands for organic fertilizers over synthetic ones driven by high preference is most evident. Advantages offered are humic acid fertilizers boosts disease resistance in plants, acts as a color enhancement and moreover ensures high nutrition in plant growth. Europe is a fast-paced growing market and reveals ideal growth trends in the international market.

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As mentioned above, 2020 being the most automotive year as there will be a lot of happenings in automotive industry ranging from launch of automatic vehicles to discussion panel for flying cars. Although, the holistic market seems to be optimal in terms of revenue, the braking units category is the most remarkable. In addition, braking systems in automatic vehicles are quite sophisticated and offer more accuracy. The trend of hydraulic braking systems is gaining much attention in the past years and is expected to reveal lasting effects on the global market. Adherence of international safety standards and certifications have a crucial role in the development of automotive disc brake market.

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According to a research study, the aerospace industry creates more jobs than any other sector. In this regards, aerospace coatings domain is a fast and emerging sector wherein it is valued at USD 2.3bn by end of 2022. Demand for technical competence, knowledge of signs and symbols in the aerospace industry and high adoption of nanotechnology in the MRO for aerospace. In addition, growing significance of nano-structure metals with low carbon footprint is expected to have a huge impact on the global market. Apart, revision of policies in the aviation industry in Asia Pacific market is expected to impart growth opportunities in the coming years.

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Market research trends in 2020 will be more data-driven and companies are willing to spend more. The deciding factor is what data they are looking and the level of input to start with, and this is possible having an insight partner around the corner. So, do let us know what you think and by leaving a comment.

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Douglas Rosenthal on Strengthening the Immune System – Patch.com

January 30th, 2020 3:42 pm

Strengthening the immunity is a very important thing. In order to be in top shape, healthy, strong, cheerful, energetic and maximally productive, we must take care of our mind and body. For our organism to work as a perfect mechanism, we must keep an eye on its parts and take care of every beat. For the perfect pulse, a resistant and healthy organism, it is necessary to provide the body with strong immunity.

The function of the immune system is to protect the body from all factors that could impair our health. With a severely damaged immune system, no one can survive despite the treatment with the most effective antibiotics. On this occasion, research scientist and structural biologist Douglas Rosenthal shares some helpful ways to strengthen your immune system and fight off disease.

Doug Rosenthal conducts research studies of the molecules that form cells and pathogens. He focuses on how pathogens infect and cause disease by producing virulence factors that target host cell molecules.

Immune system function. Why is it so important?

The immune system is activated every time a foreign body or organism is found in the human body. These are usually microorganisms (bacteria, viruses, fungi) as well as pollution particles or toxins, and we call these factors antigens. There are millions of antigens that can even have slight differences between them, which are recognized by the immune system and triggered by a response called the immune response. It is a very important task of this system to remember foreign matter and distinguish it from those belonging to the human body.

Immunity is the ability of an organism to defend itself by its mechanisms against viruses, infections, bacteria, parasites and other pathogens. As biologist Douglas Rosenthal says, there are several types of immunity, naturally acquired, artificially acquired, active and passive.

Naturally acquired immunity is one that the baby inherits from the mother, that is, antibodies that, during pregnancy, the mother transmits via the placenta to the fetus or via milk to the infant.

Artificially acquired immunity is an artificially enhanced organism to fight disease, that is, to inject antibodies that are introduced into the body, for example through vaccination.

Active immunity is created and renewed throughout life. For example, when a virus invades the body, the body creates cells that remember the body's defense response to that virus, and the next time the same virus attacks the body, the body already knows how to defend itself (healing diseases).

Passive immunity is an antibody transfer but it is not long lasting. Artificially made immunity is resorted to when there is a high risk that, for example, a particular virus will invade an organism, and there is not enough time for the organism to generate antibodies for defense itself.

How to strengthen your immunity in the best way

Above all, one should pay attention to lifestyle. Enough sleep - at least eight hours, regular physical but well-chosen activity, preferably three times a week, staying in nature, relaxing with loved ones, eliminating stress, enough water intake, recommended dose at least 8 glasses a day.

Listening to and taking care of your body is certainly a real weapon against the wicked enemies that lurk every moment of weakness to attack our bodies.

Being in harmony with immunity

According to researcher Douglas Rosenthal, the individual part must always be connected to the whole, if the whole is not right, its parts will suffer. To cure the disease is primarily to enable the body as a whole, or ideally, to function in accordance with its environment. In order to get the best recipes and guidelines, instructions and recommendations, it is necessary to monitor your body's signals and consult with a professional for further steps.

See the article here:
Douglas Rosenthal on Strengthening the Immune System - Patch.com

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Food to Boost Your Immune System and Fight the Flu – The National Herald

January 30th, 2020 3:42 pm

By Eleni Sakellis January 24, 2020

Spinach salad. Photo: By cyclonebill, via Wikimedia Commons

Foods that boost your immune system can help keep you healthy all year long, even in the midst of cold and flu season. Simple, nutrient-rich foods, full of anti-oxidants that fight free radicals support immune health. The gut is also a major part of the bodys immune system and keeping the gut healthy contributes to our overall health and well-being. A balanced diet and moderate exercise are not only good for you, they help you feel better as well, even if the long, dark winter is getting you down. Try the following recipes to help keep your immune system in top condition. For those who unfortunately do come down with cold or flu symptoms, consult your physician.

Healthy Smoothie

1 cup fresh baby spinach3/4 cup frozen green grapes1/2 cup brewed green tea, cooled1 medium ripe kiwi, peeled and chopped1 sprig fresh mint leaves1/2 medium apple, cored and chopped

Add the spinach, grapes, green tea, kiwi, mint, and chopped apple to the bowl of a blender. Blend until smooth, adding water if needed for the desired consistency.

Spinach and Feta Salad with Walnuts

1/3 cup walnuts, chopped1 lemon1/4 cup Greek extra virgin olive oil1 package fresh, pre-washed baby spinach1 Granny Smith apple, thinly sliced1/2 cup crumbled fetaGreek sea salt and freshly ground pepper to taste

Toast the walnuts in a dry pan over medium heat, stirring for about two minutes until their fragrance is released, careful not to burn them. Transfer to a plate to cool slightly. In a mixing bowl, whisk together the juice of half the lemon and the olive oil. Add the spinach, apple slices, walnuts, salt and pepper to taste and toss to coat with lemon and olive oil. Add additional lemon juice to taste, if preferred. Serve immediately.

Healthy Fruit Salad

Apples, choppedOranges, peeled and sectionedKiwis, peeled and dicedPineapple, choppedGrapesPomegranate seeds2 tablespoons finely chopped fresh mint, optionalGreek yogurt, optional

Place the cut fruits in a large bowl and mix carefully to combine. Add the grapes, cutting in half if they happen to be very large, stir and garnish the top with pomegranate seeds and fresh mint, if using. For adults only, add half or a whole bottle of white wine or champagne for special occasions, and stir. Let the fruit salad marinade for half an hour or refrigerate for up to 2 hours. Stir before serving in individual bowls, spooning some of the liquid over the top. Any leftover juices and wine make for a refreshing drink. For more Greek flavor, serve with Greek yogurt for extra protein. If preferred, top with your favorite flavor of ice cream and enjoy as a sundae.A diversity of fruits in your fruit salad adds complexity and interest, not to mention vital phytonutrients to your diet. Use about a cup of fruit per person to determine the amount of fruit you will need for your salad. The fruits listed in the recipe are suggestions, so choose your favorites and enjoy.

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Food to Boost Your Immune System and Fight the Flu - The National Herald

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‘Make sure immune system is strong and fit to fight’ – New Straits Times

January 30th, 2020 3:42 pm

KUALA LUMPUR: As international scientists and medical experts strive to find a cure and vaccine for the 2019 novel coronavirus (2019-nCov), there is also a need for Malaysians to play their part by strengthening their immune system.

Boosting the immune system would help the body fight viruses and bacterial attacks, said Malaysian Medical Association president Dr N. Ganabaskaran.

He said Malaysians should practise good eating habits and choose food that strengthened the immune system.

He said a strong immune system could reduce the risks of viral and bacteria infections.

The immune system can help the body fight infections caused by viruses and speed up the recovery process after an infection.

The immune system is the bodys defence against infectious organisms and other invaders.

The immune system is made up of a network of cells, tissues and organs that work together to protect the body.

However, Dr Ganabaskaran, said everyones immune system was different due to factors such as age, eating habits and lifestyle.

Thus, it is important to make sure that our immune system is strong and fit to fight.

One of the most important vitamins that boosts the immune system is Vitamin C.

Malaysian Medical Association president Dr N. Ganabaskaran. - NSTP/ASYRAF HAMZAH

A lack of Vitamin C can make you more prone to getting sick.

Then we have Vitamin B6, which is vital in supporting biochemical reactions in the immune system, and thirdly, Vitamin E, a powerful antioxidant that aids the body to fight off infections.

He stressed the importance of getting enough vitamins for a stronger immune system.

He said a healthy diet and adequate consumption of vitamins were essential for a strong immune system.

Consuming food that boost the immune system was encouraged in light of the influenza and 2019 n-CoV outbreak, he said.

He said food that boost the immune system included green vegetables, dairy products, kiwi fruit, lemons and oranges, as well as nuts like almonds and cashew, which were high in vitamins and antioxidants.

Even cooked poultry and shellfish are important immune boosters due to the protein and zinc they provide. It is also advisable to avoid consuming half-cooked or raw food for a while.

Shellfish that are high in zinc include crab, clams, lobster and mussels. However, observe the daily recommended amount (11mg for men and 8mg for women). Too much zinc can inhibit the immune system. Deficiencies in Vitamin D may lead to poor bone growth, cardiovascular problems and a weak immune system.

Choose supplements that contain D3 (cholecalciferol), since its good for raising your blood levels of Vitamin D.

Nutritionist Dr Sareena Hanim Hamzah said a balanced diet comprising carbohydrates, protein, fat, vitamins and minerals, as well drinking enough water, were essential to boosting the immune system.

Fruits and vegetables of various colours are important for the body to replenish and strengthen ones immunity. Fruits and vegetables contain Vitamin C, E and antioxidants that can enhance the immune system to fight against infections and pathogens.

She said citrus fruits and berries were among the best for strengthening the immune system. For vegetables, she recommended bell peppers, broccoli and spinach.

However, consumption must be in moderation.

Generally, people without any underlying health issues will not be affected by slightly higher intake, but for people who may have pre-existing health conditions or have had medical procedures done, for example to the heart, its best to consult a nutritionist for a customised regime.

Sareena said besides fruits and vegetables with high Vitamin E, a powerful antioxidant, seeds were also essential for boosting the immune system.

For instance, she said, sunflower seeds had phosphorous, magnesium and Vitamin B6.

Vitamin E is important for regulating and maintaining immune system function. Other foods with high Vitamin E include avocados, dark leafy greens and nuts.

Based on scientific research from around the world, garlic, ginger, dark chocolate and green tea are also good for the body, she said, adding that Vitamin D and probiotics from yogurt improved the immune system.

Vitamins B6 from poultry and foods with Vitamin D were recommended, she said.

Sareena urged people to practise good eating habits and stay away from processed food.

Same thing applies to high-sugar food and drinks as they may act just the opposite and accumulate unnecessary fats or lead to other medical problems.

To stay fit and have a strong immune system, people should complement a good diet with frequent or regular exercise for the best results.

Go here to read the rest:
'Make sure immune system is strong and fit to fight' - New Straits Times

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