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Global & US Gene Therapy Market 2019-2020: Immunodeficiency Syndromes – The Focus of Gene Therapy – PRNewswire

DUBLIN, Sept. 20, 2019 /PRNewswire/ -- The "Global & US Gene Therapy Market Forecast to 2020" report has been added to ResearchAndMarkets.com's offering.

Gene therapy is one of the most widely researched fields in the healthcare industry. The high potential of gene therapy to cure various chronic diseases makes it a popular research area. Various researches are being performed across the globe to explore the potential of gene therapy for the treatment of incurable diseases, such as diabetes, cancer, and HIV amongst others.

According to this new research report "Global & US Gene Therapy Market Forecast to 2020, most of the gene therapy researches are being focused on finding the treatment for cancer, followed by genetic diseases and neurological disorders, respectively. In this context, the gene therapy application chapter of the report provides a comprehensive overview of various diseases in which the gene therapy is used, along with the current and future market size of gene therapy for particular disease and its geographical break up. Primarily, the gene therapy market is dominated by oncology applications, with several companies and academic institutions focusing on novel and difficult to treat' cancers. Other therapeutic areas seeking developments in gene therapy include monogenic diseases, cardiovascular diseases, infectious diseases, inherited blindness and neurological diseases. Furthermore, the chapter also provides details regarding the various aspects of the clinical trials in the different gene therapy application by phases, vector types, etc.

The major part of the revenue of gene therapy market is generated from research phase. Most of the gene therapy products are in research phases, only few products have been commercialized till date. The report also provides the sales of major marketed gene therapy products, and the list of the products in clinical/pre-clinical research along with their clinical phases.

The companies operating in the gene therapy market are also receiving various funding, grants, and investment from government bodies and venture capitalist firms which are aiding them to develop new products. The study highlights the applications for which the investments have been received.

North America continues to have the maximum number of clinical trials in the gene therapy segment. This is a major reason for the dominant position of North America in the gene therapy market. Based on the geography, the market is divided into four regions, namely, North America, Europe, Asia, and Rest of the World. The report provides the market for each of the geography, along with its forecast till 2020.

Gene silencing, advanced therapies combining gene therapy and stem cell technology, immunodeficiency syndromes, growing interest of venture capital firms, etc. will propel the industry's growth. An analysis has also been done of a few factors limiting the growth of the industry. The report also provides insights regarding the strategies adopted by the players from 2013 to 2015 for enhancing their market share. Finally, with a view to understand the competitive landscape, the profiles of key market players have been included in the report to present a complete picture of the global gene therapy market.

Key Topics Covered:

1. Analyst View

2. Research Methodology

3. Gene Therapy - An Introduction3.1 Classification of Gene Therapy Techniques3.2 Physical Methods of Gene Transfer3.2.1 Electroporation3.2.2 Hydrodynamic3.2.3 Microinjection3.2.4 Particle Bombardment3.2.5 Ultrasound-Mediated Transfection3.3 Vectors for Gene Therapy3.3.1 Viral Vectors3.3.1.1 Adenoviral Vectors3.3.1.2 Adeno-associated Virus Vectors3.3.1.3 Retroviral Vectors3.3.1.4 Lentiviral Vectors3.3.2 Non Viral Vectors3.3.2.1 Naked DNA/Plasmid Vectors3.3.2.2 Oligonucleotides3.3.2.3 Liposomes, Lipoplexes and Polyplexes3.3.2.4 Gene-Activated Matrix

4. Industry Overview4.1 Market Drivers4.1.1 Failure of Conventional Therapies to Treat Cancers4.1.2 Rising Focus to Accelerate Commercialization of Gene Therapy in Developed Nations 4.1.3 Rising Incidence of Chronic and Life-Threatening Diseases4.1.4 Growing Interest of Venture Capital Firms4.2 Market Restraints4.2.1 Stringent Regulatory Laws & Safety Concerns 4.2.2 Challenges in Commercialization of Gene Therapy4.2.3 High Cost of the Gene Therapy Drugs4.2.4 Pitfalls in Current Technique4.2.5 Production & Manufacturing: A Unique Challenge for Gene Therapies 4.3 Market Opportunities4.3.1 Approval of Gene Therapy Drug in Europe4.3.2 Gene Therapy for Hemophilia B Offers Significant Opportunity 4.4 Industry Trends4.4.1 Gene Silencing: Gaining Momentum4.4.2 Advanced Therapies: Combining Gene Therapy and Stem Cell Technology4.4.3 Immunodeficiency Syndromes: The Focus of Gene Therapy4.4.4 Nanotechnology: Empowering Gene Therapy4.4.5 Gene Therapy: A New Hope to Treat Blindness4.4.6 Gene Therapy: Potential Cure for Cancer4.5 Winning Imperatives4.5.1 Investment in R&D Activities

5. Clinical Trial Assessment & Pipeline Analysis5.1 Clinical Trials5.1.1 By Geography5.1.2 By Indication5.1.3 By Gene Type5.1.4 By Vector5.1.5 By Clinical Trial Phase5.2 Pipeline Analysis

6. Gene Therapy Market - Regulatory Landscape & Reimbursement Scenario6.1 Regulatory Landscape6.1.1 US6.1.2 Canada6.1.3 Europe 6.1.4 Japan6.1.5 China6.1.6 India6.1.7 Australia6.2 Reimbursement Scenario in Global Market

7. Marketed Gene Therapies7.1 Neovasculgen7.2 Glybera7.3 Gendicine, Rexin-G, Oncorine7.3.1 Gendicine7.3.2 Rexin G 7.3.3 Oncorine

8. Gene Therapy Market8.1 Global8.2 US

9. Gene Therapy Market by Application9.1 Oncology9.1.1 Clinical Trials 9.1.2 Prevalence9.1.3 Market Outlook9.2 Cardiovascular Diseases9.3 Infectious Diseases9.4 Neurological Diseases9.5 Genetic Diseases9.6 Others

10. Gene Therapy Market Size by Geography10.1 North America10.2 Europe10.3 Asia10.4 Rest of the World (RoW)

11. Competitive Landscape11.1 Strategies Adopted by Various Players11.1.1 Strategic Collaborations11.1.2 Acquisitions 11.1.3 Funding & Investments11.2 Companies Progress in Gene Therapy In 2015

12. Key Players Analysis (Business Description, Clinical Pipeline, Recent Developments, Strengths & Weaknesses)12.1 Spark Therapeutics, LLC12.2 ViroMed Co. Ltd. dba VM BioPharma12.3 Advantagene Inc.12.4 Bluebird Bio12.5 Sanofi12.6 Vical Inc.12.7 Oxford BioMedica Plc12.8 Genethon12.9 uniQure N.V.12.10 Human Stem Cells Institute12.11 Shanghai Sunway Biotech Co. Ltd.12.12 Sibiono GeneTech Co. Ltd.

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Global & US Gene Therapy Market 2019-2020: Immunodeficiency Syndromes - The Focus of Gene Therapy - PRNewswire

Stem Cell-Derived Cells Market to Record an Exponential CAGR by 2025 – Commerce Gazette

Stem cell-derived cells are ready-made human induced pluripotent stem cells (iPS) and iPS-derived cell lines that are extracted ethically and have been characterized as per highest industry standards. Stem cell-derived cells iPS cells are derived from the skin fibroblasts from variety of healthy human donors of varying age and gender. These stem cell-derived cells are then commercialized for use with the consent obtained from cell donors. These stem cell-derived cells are then developed using a complete culture system that is an easy-to-use system used for defined iPS-derived cell expansion. Majority of the key players in stem cell-derived cells market are focused on generating high-end quality cardiomyocytes as well as hepatocytes that enables end use facilities to easily obtain ready-made iPSC-derived cells. As the stem cell-derived cells market registers a robust growth due to rapid adoption in stem cellderived cells therapy products, there is a relative need for regulatory guidelines that need to be maintained to assist designing of scientifically comprehensive preclinical studies. The stem cell-derived cells obtained from human induced pluripotent stem cells (iPS) are initially dissociated into a single-cell suspension and later frozen in vials. The commercially available stem cell-derived cell kits contain a vial of stem cell-derived cells, a bottle of thawing base and culture base.

The increasing approval for new stem cell-derived cells by the FDA across the globe is projected to propel stem cell-derived cells market revenue growth over the forecast years. With low entry barriers, a rise in number of companies has been registered that specializes in offering high end quality human tissue for research purpose to obtain human induced pluripotent stem cells (iPS) derived cells. The increase in product commercialization activities for stem cell-derived cells by leading manufacturers such as Takara Bio Inc. With the increasing rise in development of stem cell based therapies, the number of stem cell-derived cells under development or due for FDA approval is anticipated to increase, thereby estimating to be the most prominent factor driving the growth of stem cell-derived cells market. However, high costs associated with the development of stem cell-derived cells using complete culture systems is restraining the revenue growth in stem cell-derived cells market.

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The global Stem cell-derived cells market is segmented on basis of product type, material type, application type, end user and geographic region:

Segmentation by Product Type Stem Cell-Derived Cell Kits Stem Cell-Derived Definitive Endoderm Cell Kits Stem Cell-Derived Beta Cell Kits Stem Cell-Derived Hepatocytes Kits Stem Cell-Derived Cardiomyocytes Kits Accessories

Segmentation by End User Hospitals Research and Academic Institutions Biotechnology and Pharmaceutical Companies Contract Research Organizations/ Contract Manufacturing Organizations

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The stem cell-derived cells market is categorized based on product type and end user. Based on product type, the stem cell-derived cells are classified into two major types stem cell-derived cell kits and accessories. Among these stem cell-derived cell kits, stem cell-derived hepatocytes kits are the most preferred stem cell-derived cells product type. On the basis of product type, stem cell-derived cardiomyocytes kits segment is projected to expand its growth at a significant CAGR over the forecast years on the account of more demand from the end use segments. However, the stem cell-derived definitive endoderm cell kits segment is projected to remain the second most lucrative revenue share segment in stem cell-derived cells market. Biotechnology and pharmaceutical companies followed by research and academic institutions is expected to register substantial revenue growth rate during the forecast period.

North America and Europe cumulatively are projected to remain most lucrative regions and register significant market revenue share in global stem cell-derived cells market due to the increased patient pool in the regions with increasing adoption for stem cell based therapies. The launch of new stem cell-derived cells kits and accessories on FDA approval for the U.S. market allows North America to capture significant revenue share in stem cell-derived cells market. Asian countries due to strong funding in research and development are entirely focused on production of stem cell-derived cells thereby aiding South Asian and East Asian countries to grow at a robust CAGR over the forecast period.

Some of the major key manufacturers involved in global stem cell-derived cells market are Takara Bio Inc., Viacyte, Inc. and others.

The report covers exhaustive analysis on: Stem cell-derived cells Market Segments Stem cell-derived cells Market Dynamics Historical Actual Market Size, 2014 2018 Stem cell-derived cells Market Size & Forecast 2019 to 2029 Stem cell-derived cells Market Current Trends/Issues/Challenges Competition & Companies involved Stem cell-derived cells Market Drivers and Restraints

Regional analysis includes North America Latin America Europe East Asia South Asia Oceania The Middle East & Africa

Report Highlights: Shifting Industry dynamics In-depth market segmentation Historical, current and projected industry size Recent industry trends Key Competition landscape Strategies of key players and product offerings Potential and niche segments/regions exhibiting promising growth A neutral perspective towards market performance

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Stem Cell-Derived Cells Market to Record an Exponential CAGR by 2025 - Commerce Gazette

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.

Excerpt from:
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.

For More Details On this Report: https://www.marketstudyreport.com/reports/global-induced-pluripotent-stem-cells-ipscs-market-growth-status-and-outlook-2019-2024

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.

Thanks for reading. While I occasionally cover companies like this, my focus remains investment opportunities in liver therapeutics, specifically NASH and Cholestatic liver diseases, which are exclusive to members of my private investing community, Liver Therapy Forum Marketplace.

As a Ph.D. trained liver biomedical scientist & Scientific Consultant, I provide:

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Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

Additional disclosure: As always, my articles are meant to facilitate your understanding. Readers are expected to form their own trading plan, do their own research and take responsibility for their own actions. Investing in common stock can result in partial or total loss of capital. Please implement due diligence and invest wisely.

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