Ten-Year Bone Cancer Survivor Rides ‘Coast 2 Coast’ – Curetoday.com

Dana Vaughns and his colleagues rode 3,000 miles across the U.S. in support of funding research for the V Foundation.

BY Kristie L. Kahl

I just knew that if there was a way to get through (my cancer) and help inspire some others along the way, then I was all for it, Vaughns said. Here I am 10 years later, just completing the bike ride, which is something that I never thought I'd be able to do especially even just riding a bicycle in in the position that I would be in on a consistent basis, putting that stress on the area where I had my surgery to treat the bone cancer was a concern. But now I'm strong enough to be able to get through it and I'm very, very proud to have been able to complete it.

CURE spoke with Vaughns about his experience riding in the Coast 2 Coast 4 Cancer ride and why events like this are important to continue funding research for cancer treatments with the V Foundation.

CURE: What was running through your mind leading up to the event?Vaughns: Anxiety, a little nervousness thinking, Can I actually complete this? I mean, you could say that all the way up until the point of Now youve got to go and make it happen. And when I was out there with my other colleague, going up those hills and needing some support and some energy, I would look up, I would look at a name on someone's shirt, or even think about the names that are on my shirt that I was riding on behalf of, that really propelled me to get up every single hill that was in front of us.

Knowing what it is that I was riding for and who I was riding for, there was no way I was going to stop. I had to do it on behalf of them, I had to do it on behalf of myself. I knew that if I could complete this, then that would be a triumph over cancer for me. And for those that I was riding for, whether I was riding for them in honor of or in support of what they're going through currently right now. So, it was really just more of an inspiration for me to get through this process. And I knew that if I can do it, and those that are still here that are currently fighting cancer, they can overcome anything and that was that was a major goal for me.

What did crossing the finish line mean to you?Wow, it was it was exhilarating. I mean, to have completed 200 miles, to have climbed over 10,000 feet, to have sat on a bicycle saddle for 14 hours, to have realized that all of the training leading up to this point now had come to a conclusion was exhilarating. It was something that I'll never forget for sure.

Why do you think events like this are important?It's important because we ensure that we're staying in front of this thing called cancer. And if we can continue to rally local support, national support, do the fundraising efforts from the ground from the grassroots level in to raise funds for innovation. It's the right thing to do. And we have to find a way to be able to come up with solutions for cancer, whether they be cured, whether they be better treatments, we just have to ensure that we stay out there because the next person that may be diagnosed is going to rely on that they're going to need that. Our kids and our kids kids need to be doing this today. It is very important that we're not just doing this for now. But we're doing it for tomorrow. And that's why I think it's critically important for us to continue to do the resources necessary that can help someone when diagnosed at some point their in their lives. Know that they have a solution for what they've got going on. I think that's really critical for us to continue to do what we're doing.

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Ten-Year Bone Cancer Survivor Rides 'Coast 2 Coast' - Curetoday.com

Cancer Stem Cell Market Size Research Study including Growth Factors, Types and Application to 2025 – Daily News Collectors

The Cancer Stem Cell Market Report provides key strategies followed by leading Cancer Stem Cell industry manufactures and Sections of Market like- product specifications, volume, production value, Feasibility Analysis, Classification based on types and end user application areas with geographic growth and upcoming advancement. The Cancer Stem Cell market report provides comprehensive outline of Invention, Industry Requirement, technology and production analysis considering major factors such as Revenue, investments and business growth.

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The well-established players in the market are:

Thermo Fisher Scientific, Inc.,AbbVie, Inc.,Merck KGaA,Bionomics,LONZA,Stemline Therapeutics, Inc,Miltenyi Biotec,PromoCell GmbH,MacroGenics, Inc.,ONCOMED PHARMACEUTICALS INC,Irvine Scientific,STEMCELL Technologies Inc,Sino Biological Inc,BIOTIME, INC.

This report for Cancer Stem Cell Market discovers diverse topics such as regional market scope, product market various applications, market size according to specific product, sales and revenue by region, manufacturing cost analysis, Industrial Chain, Market Effect Factors Analysis, market size forecast, and more.

Worldwide Market Report Provides Comprehensive Analysis of:

Functional market industry outline

Up and downstream industry examination

Channels and propositions believability

Market challenge by key players

Enhancement suggestions examination

Product Type: Treatment Type,o Autologous Stem Cell Transplants,o Allogeneic Stem Cell Transplants,o Syngeneic Stem Cell Transplants,o Bone Marrow Stem Cell Transplants, Disease Type,o Breast Cancer,o Blood Cancer,o Lung Cancer,o Brain Cancer,o Colorectal Cancer,o Pancreatic Cancer,o Bladder Cancer,o Liver Cancer,o Others

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The report outlines the regulatory framework surrounding and governing numerous aspects of the market. At the end, Cancer Stem Cell industry development rival view, the industry scenario, samples, research conclusions are described. The important examination incorporated from 2014 to 2019 and till 2024 makes the report helpful assets for industry officials, promoting, sales, directors, experts, trade consultants, and others looking for key industry information with clearly given tables and charts.

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Cancer Stem Cell Market Size Research Study including Growth Factors, Types and Application to 2025 - Daily News Collectors

Current State Of Longevity Clinical Trials – Anti Aging News

Aging can be seen as one of the biggest risk factors for developing many diseases such as cancer, osteoporosis, cardiovascular disease, and Alzheimers disease. Therapies targeting aging may help to reduce suffering and benefit humanity.

Longevity drug development has evolved into an industry priority, as such a number of companies are working on developing therapies that target aging some of which are currently involved in clinical trials.

Currently there arent any longevity drugs to have received regulatory approval, although a number of therapies have passed phase I and phase II clinical human trials, there are even a few phase III trials with all of the developers racing to be the first to go to market.

Many companies working in the space of longevity drug development are more advanced than traditional biomedical companies. They are considered to be next generation because this space of research and development integrates numerous scientific and technical subdomains such as geroscience, artificial intelligence, and digital medicine. Advanced methods of diagnostics and prognostic assessments, and next generation techniques for conducting clinical trials are used by these companies compared to conventional standards which are largely based on in vitro experiments and model organisms.

Developing drugs for longevity may well be one of the more complex areas in drug discovery, as longevity focuses on prevention rather than treatment which requires complete optimization of health at the deepest levels to target biological systems that control disease.

There are 9 hallmarks of aging which are considered to be at the root causes of aging in the longevity industry, discoveries in these categories has led to clinical trials, and each trial is a step closer to ameliorating these hallmarks.

The 9 categories of longevity drugs include: genomic instability; telomere attrition; epigenetic alterations; loss of proteostasis; cellular senescence; mitochondrial dysfunction; deregulated nutrient sensing; altered intercellular communication, and stem cell exhaustion.

Longevity drugs that are currently in preclinical development include: GDF11 by Elevian; SENSOlytics by Oisin Biotechnologies; Ectopic replacement organs by Lygenesis; Egg quality restoration drugs by Jumpstart Fertility; MitoSens by SENS Research Foundation; Telomerase gene therapy, Preclinical in vivo by CNIO; Telomerase therapy and proprietary iTR technology by AgeX Therapeutics; OSKM- OCT4, Sox2, Klf, and c-Myc by Salk; OSKMLN- Oct4, Sox2, Klf4, and c-Myc LIN28, and Nanog by Turn.Bio; Lysoclear by Ichor Therapeutics; Cardizyme and Alzyme by Covalent Bioscience; Klotho by UCSF; FOXO4-DRI by Cleara Biotech; Small molecule senolytics by Antixerene; and machine learning platforms to identify peptides by Spotlight Biosciences.

Longevity drugs that have completed preclinical development include: NMD at Sinclair Lab; Gene therapy for restoration of short telomeres at Telocyte; UBX1967 at Unity Biotechnology; and Next Gen Apheresis at Conboy Lab.

Longevity drugs currently in clinical trials include: Fisetin at Mayo Clinic; Mesenchymal stem cell therapy at CRATUS; RTB101 at resTORbio; SRK-015 at Scholar Rock; SM04690 at Samumed; UBX1967 at Unity Biotechnology; J147 at Salk; NPT088 at The Michael J. Fox Foundation; Metformin at AFAR and the Albert Einstein College of Medicine; and Quercetin and Dasatinib at The Scripps Research Institute.

Longevity drugs currently in phase III clinical trials include: SM04690 at Samumed; and Targeting Aging with Metformin at the American Federation for Aging Research and the Albert Einstein College of Medicine.

Longevity drugs currently in phase II clinical trials include: UBX0101 at Unity Biotech; Quercetin and Dastinib at the Scripps Research Institute; Fisetin at the Mayo Clinic; RTB101 at resTORbio; and MSCs therapy at Cratus.

Longevity drugs currently in phase I clinical trials include: J147 at Salk; SRK-015 at Scolar Rock; and NPT088 at the Michael J. Fox Foundation and Proclara Bioscience.

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Current State Of Longevity Clinical Trials - Anti Aging News

Crigler-Najjar Syndrome Market Insights 2019-2026: Audentes Therapeutics Inc, Genethon SA, International Stem Cell Corp – US Trade Media

Crigler-Najjar Syndrome market research report has been formulated by using basic steps to conduct market research analysis which includes survey, focus groups, personal interviews, observations and field trials. This Crigler-Najjar Syndrome market report cover strategic profiling of key players in the market, comprehensively analyzing their core competencies, and drawing a competitive landscape for the market. The report provides an all-inclusive study on production capacity, consumption, import and export for all major regions across the globe. With the use of excellent resources and latest tools, this best in class Crigler-Najjar Syndrome market research report has been created to aid your business growth.

CriglerNajjar syndromeis rare genetic liver disorder characterized by abnormal accumulation of bilirubin, a yellow pigment produced by the liver. This accumulation is caused by deficiency of the enzyme called UGT1A1. This enzyme is responsible for the conversion of bilirubin into a substance that can be eliminated. CriglerNajjar Syndrome can cause significant neurological damage.

According to the statistics published in the U.S. Department of Health & Human Services, an estimated annual prevalence of Crigler-Najjar syndrome is 1 in one million newborns worldwide. Increasing research and development expenses and high demand of novel therapies are some factors fueling the market growth

Few of the major competitors currently working in the global CriglerNajjar syndrome market are International Stem Cell Corporation, Promethera, Selecta Biosciences, Inc, LOGICBIO THERAPEUTICS, INC, Genethon, Audentes Therapeutics, Teva Pharmaceutical Industries Ltd, Zydus Cadila, Endo Pharmaceuticals Inc, ANI Pharmaceuticals, Inc, Mylan N.V., Glenmark Pharmaceuticals Ltd, Amneal Pharmaceuticals LLC, Epic Pharma, LLC, Lannett and others

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Global CriglerNajjar Syndrome Market is expected to grow at a substantial CAGR in the forecast period of 2019-2026. Presence of refined healthcare infrastructure and increase in special designation from the regulatory authorities are the key factors that drive the market.

Market Drivers

Segmentation:Global CriglerNajjar Syndrome Market

By Treatment

By Drugs

By Route of Administration

By End Users

By Distribution Channel

ByGeography

Key Developments in the Market:

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Competitive Analysis:

Global CriglerNajjar syndrome market is highly fragmented and the major players have used various strategies such as new product launches, expansions, agreements, joint ventures, partnerships, acquisitions, and others to increase their footprints in this market. The report includes market shares of global CriglerNajjar syndrome market for Global, Europe, North America, Asia-Pacific, South America and Middle East & Africa.

There are 15 Chapters to display the Global Crigler-Najjar Syndrome Drug market

Chapter 1, Definition, Specifications and Classification of Crigler-Najjar Syndrome Drug , Applications of Crigler-Najjar Syndrome Drug , Market Segment by Regions;Chapter 2, Manufacturing Cost Structure, Raw Material and Suppliers, Manufacturing Process, Industry Chain Structure;Chapter 3, Technical Data and Manufacturing Plants Analysis of Crigler-Najjar Syndrome Drug , Capacity and Commercial Production Date, Manufacturing Plants Distribution, R&D Status and Technology Source, Raw Materials Sources Analysis;Chapter 4, Overall Market Analysis, Capacity Analysis (Company Segment), Sales Analysis (Company Segment), Sales Price Analysis (Company Segment);Chapter 5 and 6, Regional Market Analysis that includes United States, China, Europe, Japan, Korea & Taiwan, Crigler-Najjar Syndrome Drug Segment Market Analysis (by Type);Chapter 7 and 8, The Crigler-Najjar Syndrome Drug Segment Market Analysis (by Application) Major Manufacturers Analysis of Crigler-Najjar Syndrome Drug ;Chapter 9, Market Trend Analysis, Regional Market Trend, Market Trend by Product Type ALXN-1540, AT-342, HepaStem, Others, Market Trend by Application Hospital, Clinic, Others;Chapter 10, Regional Marketing Type Analysis, International Trade Type Analysis, Supply Chain Analysis;Chapter 11, The Consumers Analysis of Global Crigler-Najjar Syndrome Drug ;Chapter 12, Crigler-Najjar Syndrome Drug Research Findings and Conclusion, Appendix, methodology and data source;Chapter 13, 14 and 15, Crigler-Najjar Syndrome Drug sales channel, distributors, traders, dealers, Research Findings and Conclusion, appendix and data source.

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Research Methodology:Global CriglerNajjar Syndrome Market

Data collection and base year analysis is done using data collection modules with large sample sizes. The market data is analysed and forecasted using market statistical and coherent models. Also market share analysis and key trend analysis are the major success factors in the market report. To know more pleaserequest an analyst callor can drop down your enquiry.

The key research methodology used byDBMR researchteam is data triangulation which involves data mining, analysis of the impact of data variables on the market, and primary (industry expert) validation. Apart from this, other data models include Vendor Positioning Grid, Market Time Line Analysis, Market Overview and Guide, Company Positioning Grid, Company Market Share Analysis, Standards of Measurement, Top to Bottom Analysis and Vendor Share Analysis. To know more about the research methodology, drop in an inquiry to speak to our industry experts.

Primary Respondents

Demand Side: Doctors, Surgeons, Medical Consultants, Nurses, Hospital Buyers, Group Purchasing Organizations, Associations, Insurers, Medical Payers, Healthcare Authorities, Universities, Technological Writers, Scientists, Promoters, and Investors among others.

Supply Side: Product Managers, Marketing Managers, C-Level Executives, Distributors, Market Intelligence, and Regulatory Affairs Managers among others.

Reasons for Buying Crigler-Najjar Syndrome Drug market

This report provides pin-point analysis for changing competitive dynamicsIt provides a forward looking perspective on different factors driving or restraining market growthIt provides a six-year forecast assessed on the basis of how the market is predicted to growIt helps in understanding the key product segments and their futureIt provides pin point analysis of changing competition dynamics and keeps you ahead of competitorsIt helps in making informed business decisions by having complete insights of market and by making in-depth analysis of market segments

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Crigler-Najjar Syndrome Market Insights 2019-2026: Audentes Therapeutics Inc, Genethon SA, International Stem Cell Corp - US Trade Media

Global Stem Cell Antibody Market Analysis, Growth, Size, Share, Demand & Forecast 2019-2026: Thermo Fisher Scientific, Inc., Merck Group, Abcam…

Global Stem Cell Antibody Market research report has all the necessary vital details asked by the clients or any audiences in terms of market advantages or disadvantages and future market scope all mentioned in a very crystal clear manner. The report eloquently mentioned all the information regarding market competitors, growth rate, revenue ups and downs, regional players, industrial players, and applications. Even the most measly information depicting market figures are comprehensively analyzed and before being presented to the clients. The industrial players Thermo Fisher Scientific, Inc. (U.S.), Merck Group (Germany), Abcam plc (U.K.), Becton, Dickinson and Company (U.S.), Bio-Rad Laboratories, Inc. (U.S.), Cell Signaling Technology, Inc. (U.S.), Agilent Technologies, Inc. (U.S.), F. Hoffmann-La Roche Ltd (Switzerland), Danaher Corporation (U.S.), GenScript (U.S.), PerkinElmer, Inc. (U.S.), Lonza (Switzerland), and BioLegend, Inc. (U.S.) are all provided so as to make it easier for the audiences to understand the market growth rate. The current Stem Cell Antibody market research report has demonstrated all the vital market growth factors and economic fluctuations mentioned owing to the immense attention gained in recent years.

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Global Market portal aims to provide reports like these in order to draw the attention of many of the clients wanting to extrapolate some of the vital details of the Stem Cell Antibody market on a global scale. The Stem Cell Antibody market dossier talks about the market segmentation created on the basis of consensus made, product type, governments norms, key industrial players, competitive landscapes, applications, end-user, topological players, and more. The report presents a demand for individual segment in each region. It demonstrates various segments Primary Antibodies, Secondary Antibodies and sub-segments Proteomics, Drug Development, Genomics of the global Stem Cell Antibody market. The current report data simulates the market status and investment gains or losses in a very illustrative manner so as to provide the analyzed data in a very refreshed format.

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Though the paper may have certain limitations in terms of providing the information, the record has purported all the deep-seated intricate information. The clients and other readers can have all the global Stem Cell Antibody market highlights provided in this very report. The geographical regions also play an important role in enhancing the growth and development of the global Stem Cell Antibody market. The report has all the vital information regarding supply and demand, market development enhancers, market share, sales distributors, and more advocated in a very formal pattern.

There are 15 Chapters to display the Global Stem Cell Antibody market

Chapter 1, Definition, Specifications and Classification of Stem Cell Antibody , Applications of Stem Cell Antibody , Market Segment by Regions;Chapter 2, Manufacturing Cost Structure, Raw Material and Suppliers, Manufacturing Process, Industry Chain Structure;Chapter 3, Technical Data and Manufacturing Plants Analysis of Stem Cell Antibody , Capacity and Commercial Production Date, Manufacturing Plants Distribution, R&D Status and Technology Source, Raw Materials Sources Analysis;Chapter 4, Overall Market Analysis, Capacity Analysis (Company Segment), Sales Analysis (Company Segment), Sales Price Analysis (Company Segment);Chapter 5 and 6, Regional Market Analysis that includes United States, China, Europe, Japan, Korea & Taiwan, Stem Cell Antibody Segment Market Analysis (by Type);Chapter 7 and 8, The Stem Cell Antibody Segment Market Analysis (by Application) Major Manufacturers Analysis of Stem Cell Antibody ;Chapter 9, Market Trend Analysis, Regional Market Trend, Market Trend by Product Type Primary Antibodies, Secondary Antibodies, Market Trend by Application Proteomics, Drug Development, Genomics;Chapter 10, Regional Marketing Type Analysis, International Trade Type Analysis, Supply Chain Analysis;Chapter 11, The Consumers Analysis of Global Stem Cell Antibody ;Chapter 12, Stem Cell Antibody Research Findings and Conclusion, Appendix, methodology and data source;Chapter 13, 14 and 15, Stem Cell Antibody sales channel, distributors, traders, dealers, Research Findings and Conclusion, appendix and data source.

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Global Stem Cell Antibody Market Analysis, Growth, Size, Share, Demand & Forecast 2019-2026: Thermo Fisher Scientific, Inc., Merck Group, Abcam...

Induced Pluripotent Stem Cell Market Estimated to be Driven by Innovation and Industrialization – Analytics News

Overview of the iPS Cell Market with Reference to the Global Healthcare Sector Outlook

Despite the economic and political uncertainty in the recent past, the global healthcare industry has been receiving positive nudges from reformative and technological disruptions in medical devices, pharmaceuticals and biotech, in-vitro diagnostics, and medical imaging. Key markets across the world are facing a massive rise in demand for critical care services that are pushing global healthcare spending levels to unimaginable limits.

A rapidly multiplying geriatric population; increasing prevalence of chronic ailments such as cancer and cardiac disease; growing awareness among patients; and heavy investments in clinical innovation are just some of the factors that are impacting the performance of the global healthcare industry. Proactive measures such as healthcare cost containment, primary care delivery, innovation in medical procedures (3-D printing, blockchain, and robotic surgery to name a few), safe and effective drug delivery, and well-defined healthcare regulatory compliance models are targeted at placing the sector on a high growth trajectory across key regional markets.

Parent Indicators Healthcare Current expenditure on health, % of gross domestic product Current expenditure on health, per capita, US$ purchasing power parities (current prices, current PPPs) Annual growth rate of current expenditure on health, per capita, in real terms Out-of-pocket expenditure, % of current expenditure on health Out-of-pocket expenditure, per capita, US$ purchasing power parity (current prices, current PPPs) Physicians, Density per 1000 population (head counts) Nurses, Density per 1000 population (head counts) Total hospital beds, per 1000 population Curative (acute) care beds, per 1000 population Medical technology, Magnetic Resonance Imaging units, total, per million population Medical technology, Computed Tomography scanners, total, per million population

Research Methodology

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XploreMR utilizes a triangulation methodology that is primarily based on overall expenditure on life science research and R&D funding and capital equipment installed base to obtain precise market estimations and insights on life science and biotechnology associated products, technologies, applications and services. Bottom-up approach is always used to obtain insightful data for the specific country/regions. The country-specific data is again analysed to derive data at a global level. This methodology ensures high quality and accuracy of information.

Secondary research is used at the initial phase to identify the feasibility of the target products/technology categories and its respective segments, product and service offerings, equipment installed base in end use facilities, adoption rate and future impact of new technologies. Additionally, per capita consumption of kits, reagents and consumables among end users is tracked at a granular level to obtain the most accurate information. Each piece of information is eventually analysed during the entire research project which builds a strong base for the primary research information.

Primary research participants include demand-side respondents such as laboratory managers, procurement managers, research supervisors at academic and research institutes, as well as key opinion leaders in addition to supply-side respondents such as equipment and reagent manufacturers, custom solution and service providers who provide valuable insights on trends, research application of products and technologies, purchasing patterns, services offered and associated pricing.

Quantitative and qualitative assessment of basic factors driving demand, economic factors/cycles and growth rates and strategies utilized by key players in the market is analysed in detail while forecasting, in order to project year-on-year growth rates. These Y-o-Y growth projections are checked and aligned as per associated industry/product lifecycles and further utilized to develop market numbers at a holistic level.

On the other hand, we also analyse annual reports of various companies, investor presentations, SEC filings, 10k reports and earning call transcripts operating in the market to fetch substantial information about the market size, trends, opportunity, drivers, restraints and to analyse key players and their market shares. Key companies are segmented at tier-level based on their revenues, product portfolio and presence.

Please note that these are the partial steps that are being followed while developing the market size. Besides this, forecasting will be done based on our internal proprietary model which also uses different macro-economic factors such as overall life science research expenditure, R&D funding, industry based demand driving factors impacting the market and its forecast trends apart from other macroeconomic factors.

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Standard Report Structure Executive Summary Market Definition Macro-economic analysis Parent Market Analysis Market Overview Forecast Factors Segmental Analysis and Forecast Regional Analysis Competition Analysis

Market Taxonomy

The global iPS cell market has been segmented into:

Cell Type Hepatocytes Fibroblasts Keratinocytes Neurons Others

Application Drug Development Regenerative Medicine Toxicity Testing

End User Academic and Research Institutes Biotechnology Companies

Region North America Latin America Europe Asia Pacific excluding China (APEC) China Middle East and Africa (MEA)

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Induced Pluripotent Stem Cell Market Estimated to be Driven by Innovation and Industrialization - Analytics News

2024 Projections: Induced Pluripotent Stem Cells (iPSCs) Market Report by Type, Application and Regional Outlook – TheSlapClap

The Induced Pluripotent Stem Cells (iPSCs) market report added recently by Market Study Report, LLC, evaluates the industry in terms of market size, market share, revenue estimation, and geographical outlook. The study also delivers a precise summary that illustrates the competitive milieu, growth opportunities and application landscape of the Induced Pluripotent Stem Cells (iPSCs) market depending on the industrys financial and non-financial impact.

The recent report about the Induced Pluripotent Stem Cells (iPSCs) market is a detailed synopsis of the projections of this business space in tandem with an evaluation of the industry segmentation. The report depicts the Induced Pluripotent Stem Cells (iPSCs) market to evolve as one of most profitable verticals, procuring substantial valuation by the end of the estimated duration, while simultaneously registering a profitable growth rate over the forecast timespan. The expansion opportunities that are prevalent in this business alongside the industrys geographical reach have also been stated in the report.

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An inherent overview of this report:

Recognizing the basic business drivers and challenges:

Unveiling the geographical landscape of this market:

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Describing the competitive spectrum of the Induced Pluripotent Stem Cells (iPSCs) market:

A succinct outline of the Induced Pluripotent Stem Cells (iPSCs) market segmentation

. Further, the report mentions specifics about the product market share as well as the remuneration to be accumulated by every type.

. Additionally, the study projects every application segments valuation as well as current market share.

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Some of the Major Highlights of TOC covers:

Executive Summary

Manufacturing Cost Structure Analysis

Development and Manufacturing Plants Analysis of Induced Pluripotent Stem Cells (iPSCs)

Key Figures of Major Manufacturers

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2024 Projections: Induced Pluripotent Stem Cells (iPSCs) Market Report by Type, Application and Regional Outlook - TheSlapClap

Dicerna And DCR-A1AT In Alpha-1 Antitrypsin Deficiency-Associated Liver Disease – Seeking Alpha

Introduction

Dicerna Pharmaceuticals (DRNA) is a small-cap ($980M) developing RNA interference (RNAi) based therapeutics for rare and/or chronic diseases affecting the kidney and liver.

The mechanism of RNAi was first described in the late 1990s by Drs. Andrew Fire and Craig Mello. In 2006, the Nobel Prize community acknowledged the paradigm-changing seminal concept by jointly awarding both scientists the 2006 Nobel Prize for Physiology or Medicine. Their mechanism of degrading mRNA from a specific gene proposed that:

RNA interference is activated when RNA molecules occur as double-stranded pairs in the cell. Double-stranded RNA activates biochemical machinery which degrades those mRNA molecules that carry a genetic code identical to that of the double-stranded RNA. When such mRNA molecules disappear, the corresponding gene is silenced and no protein of the encoded type is made.

Two decades following the seminal mechanistic discovery, the first RNAi based therapeutics, Onpattro (patisiran) by Alnylam Pharmaceuticals (ALNY) was approved by the FDA in 2018. To understand how competitive this technology could become, look no further than the 2017 legal tussle over RNAi trade secrets between Alnylam and Dicerna. Apparently, this issue has now been resolved with both sides claiming the usual "no wrongdoing".

Dicerna has created a proprietary RNAi technology platform, GalXC, "a next-generation RNAi-based therapies designed to silence disease-driving genes in the liver. GalXC-based therapies are processed by the Dicer enzyme, which is the natural initiation point for RNAi within the human cell. By using the Dicer enzyme as the entry point into the RNAi, we seek to optimize the activity of the RNAi pathway so that it operates in the most specific and potent fashion. Compounds produced via GalXC are intended to be broadly applicable across multiple therapeutic areas, including rare diseases, viral infectious diseases, chronic liver diseases, and cardiovascular diseases."

It has a diverse pipeline with drug candidates in preclinical studies and at different phases of clinical development. The most advanced pipeline, DCR-PHXC, a breakthrough drug designate, is in Phase 3 study for the Primary Hyperoxaluria, a potential rare end-stage kidney disease, which is characterized by the recurrent kidney and bladder stones. Other drug candidates are DCR-HBVS and DCR-A1AT for chronic hepatitis B infection and alpha-1 antitrypsin deficiency-associated (A1AT) liver disease, respectively.

Alpha-1 antitrypsin deficiency (A1AT) is a genetic disorder that affects the liver and lungs. A1AT is caused by a mutation in the SERPINA1 gene. Alpha-1 antitrypsin protein regulates the effects of neutrophil elastase, an enzyme released from white blood cells to fight infection. Neutrophil elastase can induce chronic uninhibited tissue breakdown in the lung alveoli if not tightly controlled by alpha-1 antitrypsin.

Abnormal alpha-1 antitrypsin can also accumulate in the liver causing damage. A1AT can present from birth to old age and is the most frequent cause of metabolic liver disease in pediatric patients and the second most common indication for liver transplantation after biliary atresia. Suggesting that Alpha-1 antitrypsin induces protective effects in the lungs and liver against damage. Exposure to tobacco smoke, chemicals, and dust has been proposed to impact the severity of A1AT.

NIH notes that:

10% of infants with A1AT develop liver disease, which often causes yellowing of the skin and whites of the eyes (jaundice). Approximately 15% of adults with A1AT develop liver damage (cirrhosis) due to the formation of scar tissue in the liver.

The disorder affects about 120,000 European individuals or 1 in 1,500 to 3,500 individuals with European ancestry with many more being undiagnosed, particularly people with a lung condition called chronic obstructive pulmonary disease.

Therapeutics: Four alpha-1 antitrypsin products derived from a human plasma Prolastin, Zemaira, Glassia, and Aralast are approved by the FDA as intravenous augmentation A1AT therapy, costing up to $100,000 annually per patient. Alternative strategies currently being investigated, includes new delivery strategies, the use of gene therapy or Induced pluripotent stem cells (iPSCs), and silencing RNA strategies.

In Q3/2019, DRNA announced:

Initiation of a multi-center Phase 1/2 trial of DCR-A1AT is expected in the third quarter of 2019. The proposed parallel-group, placebo-controlled study will evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of DCR-A1AT in adult healthy volunteers (HVs) and patients with A1AT deficiency-associated liver disease.

At the end of Q2/2019, DRNA reported that cash and cash equivalents were $345.3M. DRNA perceives it has sufficient funds to execute current clinical trials and other operating expenses beyond 2020. DRNA has ongoing collaborative license agreements with Eli Lilly (LLY), Alexion (ALXN) and Boehringer Ingelheim International.

With 3 clinical-stage programs, several catalytic events are expected in 2020 and beyond.

RNAi-based therapeutics are high risk due to potential safety and tolerability signals. In 2016, ALNY halted further clinical development of Phase 3 drug candidate, revusiran, for treating a rare and fatal disorder called ATTR amyloidosis with cardiomyopathy, due to safety signals.

It has been proposed that the GalXC-Dicer enzyme RNAi platform invented by DRNA is designed to use the lowest possible dose whilst providing therapeutic efficacy and improved safety profile. Additional risks that could negatively impact the share price are negative data readout from clinical trials.

Institutional ownership currently stands at 82.35%, with 116 institutional holders accounting for 56,295,099 total shares. Analysts recommend a strong buy with a 12-month consensus price target of $22.25.

CEO & President Doug Fambrough on executing DRNA clinical and financial strategy:

The first aspect of the strategy is to go deep on select opportunities addressing a high unmet medical need with what we believe is a high probability of clinical and commercial success.

Our internal clinical pipeline reflects these choices including two rare diseases, primary hyperoxaluria and alpha-1 antitrypsin deficiency-associated liver disease, where we plan to drive development and commercialization either wholly or largely on our own and one prevalent disease, chronic hepatitis B virus infection, where we are seeking a development and commercialization partner, concomitant with Phase 1 proof-of-concept data.

The second aspect of the strategy is to realize the potential of our technology against all remaining targets through collaboration and discovery stage licensing with therapeutic area leaders. Our collaborations with Eli Lilly, Alexion and Boehringer Ingelheim reflect this aspect of our strategy. It is our expectation and plan that we will expand on both aspects of the strategy in coming quarters, both expanding our internal pipeline and expanding our circle of corporate collaborators, funding for much will help us drive the internal pipeline.

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Dicerna And DCR-A1AT In Alpha-1 Antitrypsin Deficiency-Associated Liver Disease - Seeking Alpha

Angelman Foundation Honors 4 for Their Research, Advocacy Efforts – Angelman Syndrome News

The Angelman Syndrome Foundation (ASF) recently honored four individuals for their exceptional contributions to the Angelman syndrome community and to advancing research.

Awards were presented to a special education specialist, researcher, former executive director of the foundation, and geneticist, during the 2019 ASF Scientific Symposium and Family Conference, an event that brought together families, educators, care providers, physicians, scientists, and other stakeholders to learn and discuss the latest on Angelman syndrome (AS).

Erin Sheldon was given the Harry & Audrey Angelman Award for Meritorious Service, an honor bestowed to those who demonstrate a strong commitment to enhancing disease awareness and understanding in their respective community, the ASF announced.

The mother of a daughter with Angelman, Sheldon has long supported AS families, advocating for inclusive schools and communities. Angelman is a complex neurological disorder that causes intellectual disabilities and issues with speech, among other symptoms. For her masters degree in education, Sheldon focused on the learning needs of students with complex disabilities.

The Dr. Claudia Benton Award for Scientific Research was presented to Stormy Chamberlain, PhD, a widely published researcher in AS and UBE3A, the protein coding gene associated with Angelman. She is an ASF board member who has made many presentations on the topic.

Chamberlain established a lab at the University of Connecticut, where shes an assistant professor in the genetics and developmental biology department, and assistant director of the graduate program in genetics and developmental biology. In her research, sheused induced pluripotent stem cells (IPSCs) to model and study genetic imprinting implicated in Angelman and other disorders.

Of note, IPSCs are derived from either skin or blood cells that have been reprogrammed back into a stem cell-like state, which allows for the development of an unlimited source of any type of human cell thats needed for therapeutic purposes. Genetic imprinting is a phenomenon that occurs when only one of the two copies of a given gene (one copy is inherited from each parent) is turned on, depending on the parent it originated from.

Receiving the ASFs Lifetime Achievement Award was Eileen Braun, who had been the foundations executive director from 2004 until this past spring. This award has now been renamed in her honor. The graduate of the Mayo Clinic School of Health Sciences, Braun has worked as a registered respiratory therapist at several medical centers, including the Mayo Clinic. She has four children, one of whom has Angelman, and currently serves as the ASFs director of operations.

During her time as ASF executive director, Braun started the ASF Walk fundraiser and organized the foundations first research roadmap (a plan that prioritized research investment toward improved patient care), and boosted research investment from $275,000 to more than $10 million. Braun also oversaw development of 12 international AS clinics and the worlds largest AS registry, and hosted 19 conferences and symposiums.

The first Eileen Braun Lifetime Achievement Award was given to Arthur Beaudet, MD, for his decades-long contribution to the fields of mammalian genetics and Angelman. A Yale University graduate and chair of molecular and human genetics at the Baylor College of Medicine, Beaudet and other scientists in 1997 identified the Angelman gene UBE3A. In 2015, his lab collaboratively showed that oligonucleotides could be used to activate the paternal copy of Angelman in mice, a promising step toward AS treatment.

A former National Institutes of Health research associate, Beaudet is a prolific author who served as president of the American Society of Human Genetics, and was elected to the National Academy of Medicine and National Academy of Sciences.

The board and staff of the Angelman Syndrome Foundation, as well as the entire community of families, teachers and scientists are grateful to these four individuals for their dedication and hard work, the organization said.

The ASF works to advance the awareness and treatment of AS through education, research, and patient and family support. Angelman syndrome occurs in about 1 in 15,000 live births.

Mary M. Chapman began her professional career at United Press International, running both print and broadcast desks. She then became a Michigan correspondent for what is now Bloomberg BNA, where she mainly covered the automotive industry plus legal, tax and regulatory issues. A member of the Automotive Press Association and one of a relatively small number of women on the car beat, Chapman has discussed the automotive industry multiple times of National Public Radio, and in 2014 was selected as an honorary judge at the prestigious Cobble Beach Concours dElegance. She has written for numerous national outlets including Time, People, Al-Jazeera America, Fortune, Daily Beast, MSN.com, Newsweek, The Detroit News and Detroit Free Press. The winner of the Society of Professional Journalists award for outstanding reporting, Chapman has had dozens of articles in The New York Times, including two on the coveted front page. She has completed a manuscript about centenarian car enthusiast Margaret Dunning, titled Belle of the Concours.

The rest is here:
Angelman Foundation Honors 4 for Their Research, Advocacy Efforts - Angelman Syndrome News

Donations For OVC Lameness Research – Standardbred Canada

Published: September 19, 2019 2:38 pm ET

A team of equine veterinarians and researchers from the Ontario Veterinary College (OVC) are excited to announce clinical trials are ready to begin using allogeneic umbilical cord blood stem cells in the treatment of superficial digital flexor tendonitis or bowed tendons in horses.

The goal is to develop a readily available treatment to prevent the formation of scar tissue and create good quality tendon tissue to replace the damaged tissues, which are less prone to re-injury. The team includes Dr. Thomas Koch, Dr. Nathalie Cot, Dr. Stephanie Nykamp, graduate student Alejandro Merchan Munoz, led by primary investigator Dr. Judith Koenig.

Many thanks goes to Ontario Equestrian (OE) and its members who scrapped their membership cards to create the Member Equine Research Fund program in 2017. This year, OE members chose to donate to lameness studies resulting in a donation of just over $30,000. The Equinosis Q Lameness Locator , previously donated to the OVC by the Equine Foundation of Canada (EFC) will play an integral role in this study. Funding has also been provided by Equine Guelph.

There is a great demand in the industry for more efficient treatment options for superficial digital tendonitis, improved recovery, and prevention of re-injury. Currently, the re-injury rates reported are between 56 to 75 per cent for superficial digital flexor tendon injuries once the horses return to competition, shares Dr. Koenig.

Cultured bone marrow derived stem cell treatments have been found to be effective and also boasts a low re-injury rate, but there is a short time frame for this treatment to be effective. A study out of England found that the tendon needs to be injected within six weeks of an injury in order to be beneficial, however this can be hard to accomplish depending on the expansion time needed to harvest the bone marrow.

In a pilot study conducted by Dr. Koenig with three horses, it was found that repeated injections of the allogeneic stem cells were very effective and that two of the horses were able to successfully return to racing and the third was able to start a career as a competitive sport horse.

My hope is that we can develop a treatment that can be available within an hour of taking out of the freezer, says Koenig. This treatment uses allogeneic stem cells, which means the stem cells do not need to be from the recipient. These stem cells are collected non-invasively from the umbilical cords of newborn foals and then stored for later use. See video and prior research leading up to these clinical trials.

The next challenge is to enroll horses in this study. The study requires very tight parameters in order to have meaningful results, says Koenig. The horses need to be Thoroughbreds in race training that have raced at least three times and incurred an injury to their superficial digital flexor tendon (SDFT) and have sustained lesions that have been diagnosed by a veterinarian. The lesions also will need to fall between specific measurements. The treatment and rehab period is expected to take between eight months to one year. The owners will receive cost incentives for enrolling their horses and following treatment and rehab protocols (more details on enrollment).

A lameness evaluation will be performed followed by an assessment using the Equinosis Q Lameness Locator . The Lameness Locator, which is the current gold standard for semi quantitative field measurements of lameness, will be used as an additional objective evaluation to confirm the diagnosis. The Lameness Locator will prevent any biases as it does not know what study group the horse is in (treatment or control). Initial blood work will be done to evaluate baseline immune status. Comparisons in blood work will be performed over the treatment period. The person that is carrying out all these tests will be blind to which study group the horse is in. A strict exercise and rehab protocol will be provided. Each horses progress will be monitored at monthly intervals.

It is the hopes of the study that the horses will return to full work within eight months to a year. The progress of the horses in the stem cell treatment group will be compared to that of a control group which will be receiving platelet rich plasma (PRP) injections. PRP is currently a common treatment used in the field for superficial digital flexor tendonitis.

Having more efficient and effective treatment options for superficial digital flexor tendonitis will improve the health of the horse, extend competitive careers, and reduce economic losses. This clinical trial will provide insight on the very promising treatment of allogeneic umbilical cord blood stem cells. Thanks to industry support from Ontario Equestrian and its members, Equine Foundation of Canada, and Equine Guelph this clinical trial is imminent and may bring the industry one step closer to providing optimal SDFT treatment options for our equine partners.

(Equine Guelph)

Link:
Donations For OVC Lameness Research - Standardbred Canada