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    Spinocerebellar Ataxia (SCA) Pipeline Review H1 2017

    Spinocerebellar Ataxia (SCA) Pipeline Review H1 2017

    • Report Code ID: RW0001799287
    • Category Pharmaceuticals
    • No. of Pages 60
    • Publication Month May-17
    • Publisher Name Global Markets Direct
    Spinocerebellar Ataxia (SCA) - Pipeline Review, H1 2017

    Summary

    Publisher's latest Pharmaceutical and Healthcare disease pipeline guide Spinocerebellar Ataxia (SCA) - Pipeline Review, H1 2017, provides an overview of the Spinocerebellar Ataxia (SCA) (Genetic Disorders) pipeline landscape.

    Spinocerebellar ataxias (SCAs) are a group of inherited conditions that affect the brain and spinal cord causing progressive difficulty with coordination. Symptoms include poor coordination, unsteady walk and a tendency to stumble, change in speech and difficulty swallowing.

    Report Highlights

    Publisher's Pharmaceutical and Healthcare latest pipeline guide Spinocerebellar Ataxia (SCA) - Pipeline Review, H1 2017, provides comprehensive information on the therapeutics under development for Spinocerebellar Ataxia (SCA) (Genetic Disorders) , complete with analysis by stage of development, drug target, mechanism of action (MoA) , route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.

    The Spinocerebellar Ataxia (SCA) (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Spinocerebellar Ataxia (SCA) and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase III, Phase II, Phase I, Preclinical and Discovery stages are 2, 3, 1, 4 and 1 respectively. Similarly, the Universities portfolio in Phase III, Preclinical and Discovery stages comprises 1, 1 and 2 molecules, respectively.

    Spinocerebellar Ataxia (SCA) (Genetic Disorders) pipeline guide helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage. The guide is built using data and information sourced from Publisher's proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

    Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

    Scope

    - The pipeline guide provides a snapshot of the global therapeutic landscape of Spinocerebellar Ataxia (SCA) (Genetic Disorders) .
    - The pipeline guide reviews pipeline therapeutics for Spinocerebellar Ataxia (SCA) (Genetic Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources.
    - The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.
    - The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.
    - The pipeline guide reviews key companies involved in Spinocerebellar Ataxia (SCA) (Genetic Disorders) therapeutics and enlists all their major and minor projects.
    - The pipeline guide evaluates Spinocerebellar Ataxia (SCA) (Genetic Disorders) therapeutics based on mechanism of action (MoA) , drug target, route of administration (RoA) and molecule type.
    - The pipeline guide encapsulates all the dormant and discontinued pipeline projects.
    - The pipeline guide reviews latest news related to pipeline therapeutics for Spinocerebellar Ataxia (SCA) (Genetic Disorders)

    Reasons to buy

    - Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
    - Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.
    - Find and recognize significant and varied types of therapeutics under development for Spinocerebellar Ataxia (SCA) (Genetic Disorders) .
    - Classify potential new clients or partners in the target demographic.
    - Develop tactical initiatives by understanding the focus areas of leading companies.
    - Plan mergers and acquisitions meritoriously by identifying key players and it's most promising pipeline therapeutics.
    - Formulate corrective measures for pipeline projects by understanding Spinocerebellar Ataxia (SCA) (Genetic Disorders) pipeline depth and focus of Indication therapeutics.
    - Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope.
    - Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline.
    Table of Contents
    List of Tables
    List of Figures
    Introduction
    Publisher Report Coverage
    Spinocerebellar Ataxia (SCA) - Overview
    Spinocerebellar Ataxia (SCA) - Therapeutics Development
    Pipeline Overview
    Pipeline by Companies
    Pipeline by Universities/Institutes
    Products under Development by Companies
    Products under Development by Universities/Institutes
    Spinocerebellar Ataxia (SCA) - Therapeutics Assessment
    Assessment by Target
    Assessment by Mechanism of Action
    Assessment by Route of Administration
    Assessment by Molecule Type
    Spinocerebellar Ataxia (SCA) - Companies Involved in Therapeutics Development
    Bio Blast Pharma Ltd
    Ionis Pharmaceuticals Inc
    Reata Pharmaceuticals Inc
    Shionogi & Co Ltd
    Shire Plc
    Spark Therapeutics Inc
    Vybion Inc
    Spinocerebellar Ataxia (SCA) - Drug Profiles
    2AU-2 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Antisense RNAi Oligonucleotide to Inhibit Ataxin-1 for Spinocerebellar Ataxia - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Antisense RNAi Oligonucleotide to Inhibit Ataxin-7 for Spinocerebellar Ataxia Type 7 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    ASO-7 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    BHV-4157 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    epoetin alfa - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    immune globulin (human) - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    INT-41 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    JOT-105 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    riluzole - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    RNAi Gene Therapy to Inhibit Ataxin-3 for Machado-Joseph Disease - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    rovatirelin - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    RTA-901 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Stem Cell Therapy for Diabetes Mellitus, Acute Liver Failure, Osteoarthritis and Neurodegenerative Diseases - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    trehalose - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Spinocerebellar Ataxia (SCA) - Dormant Projects
    Spinocerebellar Ataxia (SCA) - Product Development Milestones
    Featured News & Press Releases
    Jan 26, 2017: Bioblast Pharma to Host Key Opinion Leader Lunch Focused on Orphan Neurological Diseases on February 2
    Jan 18, 2017: Bioblast Announces Phase 2a Results of Trehalose in Patients with Spinocerebellar Ataxia Type 3 (SCA3)
    Jun 01, 2016: Scripps Florida Scientists Create Compound that Erases Disease-Causing RNA Defects
    May 27, 2016: Announcement of Additional Clinical Trial of "KPS-0373 " for the Treatment of Spinocerebellar Ataxia
    Mar 03, 2016: Portage Biohaven orphan drug designation request granted for the treatment of spinocerebellar ataxia
    Sep 14, 2015: BioBlast Granted Patent From USPTO for Cabaletta for the Treatment of Spinocerebellar Ataxia
    Jun 29, 2015: BioBlast Pharma Receives Orphan Drug Designation From the European Commission for Cabaletta for the Treatment of Spinocerebellar Ataxia
    Mar 30, 2015: BioBlast Pharma Announces Positive in vivo Preclinical Proof-of-Concept Results of Its Cabaletta Platform in Spinocerebellar Ataxia Type 3
    Nov 19, 2014: Bio Blast Pharma Receives Orphan Drug Designation From U.S. FDA for Cabaletta for the Treatment of Spinocerebellar Ataxia Type 3
    Appendix
    Methodology
    Coverage
    Secondary Research
    Primary Research
    Expert Panel Validation
    Contact Us
    Disclaimer

    List of Tables

    Number of Products under Development for Spinocerebellar Ataxia (SCA) , H1 2017
    Number of Products under Development by Companies, H1 2017
    Number of Products under Development by Universities/Institutes, H1 2017
    Products under Development by Companies, H1 2017
    Products under Development by Universities/Institutes, H1 2017
    Number of Products by Stage and Target, H1 2017
    Number of Products by Stage and Mechanism of Action, H1 2017
    Number of Products by Stage and Route of Administration, H1 2017
    Number of Products by Stage and Molecule Type, H1 2017
    Spinocerebellar Ataxia (SCA) - Pipeline by Bio Blast Pharma Ltd, H1 2017
    Spinocerebellar Ataxia (SCA) - Pipeline by Ionis Pharmaceuticals Inc, H1 2017
    Spinocerebellar Ataxia (SCA) - Pipeline by Reata Pharmaceuticals Inc, H1 2017
    Spinocerebellar Ataxia (SCA) - Pipeline by Shionogi & Co Ltd, H1 2017
    Spinocerebellar Ataxia (SCA) - Pipeline by Shire Plc, H1 2017
    Spinocerebellar Ataxia (SCA) - Pipeline by Spark Therapeutics Inc, H1 2017
    Spinocerebellar Ataxia (SCA) - Pipeline by Vybion Inc, H1 2017
    Spinocerebellar Ataxia (SCA) - Dormant Projects, H1 2017

    List of Figures

    Number of Products under Development for Spinocerebellar Ataxia (SCA) , H1 2017
    Number of Products under Development by Companies, H1 2017
    Number of Products under Development by Universities/Institutes, H1 2017
    Number of Products by Targets, H1 2017
    Number of Products by Stage and Targets, H1 2017
    Number of Products by Top 10 Mechanism of Actions, H1 2017
    Number of Products by Stage and Top 10 Mechanism of Actions, H1 2017
    Number of Products by Routes of Administration, H1 2017
    Number of Products by Stage and Routes of Administration, H1 2017
    Number of Products by Molecule Types, H1 2017
    Number of Products by Stage and Molecule Types, H1 2017
    Bio Blast Pharma Ltd
    Ionis Pharmaceuticals Inc
    Reata Pharmaceuticals Inc
    Shionogi & Co Ltd
    Shire Plc
    Spark Therapeutics Inc
    Vybion Inc

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