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    Spinocerebellar Ataxia (SCA) - Pipeline Review, H2 2016

    Spinocerebellar Ataxia (SCA) - Pipeline Review, H2 2016

    • Report Code ID: RW0001499736
    • Category Pharmaceuticals
    • No. of Pages 65
    • Publication Month Nov-16
    • Publisher Name Global Markets Direct
    Spinocerebellar Ataxia (SCA) - Pipeline Review, H2 2016

    Summary

    Publisher's latest Pharmaceutical and Healthcare disease pipeline guide Spinocerebellar Ataxia (SCA) - Pipeline Review, H2 2016, 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, H2 2016, 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 1, 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).

    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 5
    List of Figures 5
    Introduction 6
    Publisher Report Coverage 6
    Spinocerebellar Ataxia (SCA) Overview 7
    Therapeutics Development 8
    Pipeline Products for Spinocerebellar Ataxia (SCA) - Overview 8
    Pipeline Products for Spinocerebellar Ataxia (SCA) - Comparative Analysis 9
    Spinocerebellar Ataxia (SCA) - Therapeutics under Development by Companies 10
    Spinocerebellar Ataxia (SCA) - Therapeutics under Investigation by Universities/Institutes 11
    Spinocerebellar Ataxia (SCA) - Pipeline Products Glance 12
    Late Stage Products 12
    Clinical Stage Products 13
    Early Stage Products 14
    Spinocerebellar Ataxia (SCA) - Products under Development by Companies 15
    Spinocerebellar Ataxia (SCA) - Products under Investigation by Universities/Institutes 16
    Spinocerebellar Ataxia (SCA) - Companies Involved in Therapeutics Development 17
    Bio Blast Pharma Ltd 17
    Biohaven Pharmaceutical Holding Company Limited 18
    Ionis Pharmaceuticals Inc 19
    Shionogi & Co Ltd 20
    Shire Plc 21
    Spark Therapeutics Inc 22
    Vybion Inc 23
    Spinocerebellar Ataxia (SCA) - Therapeutics Assessment 24
    Assessment by Monotherapy Products 24
    Assessment by Target 25
    Assessment by Mechanism of Action 27
    Assessment by Route of Administration 29
    Assessment by Molecule Type 31
    Drug Profiles 33
    2AU-2 - Drug Profile 33
    Product Description 33
    Mechanism Of Action 33
    R&D Progress 33
    Antisense Oligonucleotides to Inhibit ATXN2 for Spinocerebellar Ataxia - Drug Profile 34
    Product Description 34
    Mechanism Of Action 34
    R&D Progress 34
    Antisense RNAi Oligonucleotide to Inhibit Ataxin-1 for Spinocerebellar Ataxia - Drug Profile 35
    Product Description 35
    Mechanism Of Action 35
    R&D Progress 35
    Antisense RNAi Oligonucleotide to Inhibit Ataxin-7 for Spinocerebellar Ataxia Type 7 - Drug Profile 36
    Product Description 36
    Mechanism Of Action 36
    R&D Progress 36
    BHV-4157 - Drug Profile 37
    Product Description 37
    Mechanism Of Action 37
    R&D Progress 37
    epoetin alfa - Drug Profile 39
    Product Description 39
    Mechanism Of Action 39
    R&D Progress 39
    immune globulin (human) - Drug Profile 40
    Product Description 40
    Mechanism Of Action 40
    R&D Progress 40
    INT-41 - Drug Profile 44
    Product Description 44
    Mechanism Of Action 44
    R&D Progress 44
    JOT-105 - Drug Profile 45
    Product Description 45
    Mechanism Of Action 45
    R&D Progress 45
    riluzole - Drug Profile 46
    Product Description 46
    Mechanism Of Action 46
    R&D Progress 46
    RNAi Gene Therapy to Inhibit Ataxin-3 for Machado-Joseph Disease - Drug Profile 49
    Product Description 49
    Mechanism Of Action 49
    R&D Progress 49
    rovatirelin - Drug Profile 50
    Product Description 50
    Mechanism Of Action 50
    R&D Progress 50
    Stem Cell Therapy for Osteoarthritis, Diabetes Mellitus, Acute Liver Failure and Neurodegenerative Diseases - Drug Profile 51
    Product Description 51
    Mechanism Of Action 51
    R&D Progress 51
    trehalose - Drug Profile 52
    Product Description 52
    Mechanism Of Action 52
    R&D Progress 52
    Spinocerebellar Ataxia (SCA) - Dormant Projects 56
    Spinocerebellar Ataxia (SCA) - Product Development Milestones 57
    Featured News & Press Releases 57
    Jul 20, 2016: Biohaven Reports Dosing Of First Subject In Pharmacokinetic And Safety Trial Of BHV-4157 57
    Jul 05, 2016: Biohaven Receives FDA Investigational New Drug Application (IND) Approval for BHV-4157 57
    Jun 01, 2016: Scripps Florida Scientists Create Compound that Erases Disease-Causing RNA Defects 58
    May 27, 2016: Announcement of Additional Clinical Trial of "KPS-0373 " for the Treatment of Spinocerebellar Ataxia 59
    May 25, 2016: Portage Announces Orphan Drug Designation Request Granted to Biohaven for BHV-4157 59
    Mar 03, 2016: Portage Biohaven orphan drug designation request granted for the treatment of spinocerebellar ataxia 60
    Sep 14, 2015: BioBlast Granted Patent From USPTO for Cabaletta for the Treatment of Spinocerebellar Ataxia 61
    Jun 29, 2015: BioBlast Pharma Receives Orphan Drug Designation From the European Commission for Cabaletta for the Treatment of Spinocerebellar Ataxia 61
    Mar 30, 2015: BioBlast Pharma Announces Positive in vivo Preclinical Proof-of-Concept Results of Its Cabaletta Platform in Spinocerebellar Ataxia Type 3 62
    Nov 19, 2014: Bio Blast Pharma Receives Orphan Drug Designation From U.S. FDA for Cabaletta for the Treatment of Spinocerebellar Ataxia Type 3 62
    Appendix 64
    Methodology 64
    Coverage 64
    Secondary Research 64
    Primary Research 64
    Expert Panel Validation 64
    Contact Us 64
    Disclaimer 65

    List of Tables

    Number of Products under Development for Spinocerebellar Ataxia (SCA), H2 2016 8
    Number of Products under Development for Spinocerebellar Ataxia (SCA) - Comparative Analysis, H2 2016 9
    Number of Products under Development by Companies, H2 2016 10
    Number of Products under Investigation by Universities/Institutes, H2 2016 11
    Comparative Analysis by Late Stage Development, H2 2016 12
    Comparative Analysis by Clinical Stage Development, H2 2016 13
    Comparative Analysis by Early Stage Development, H2 2016 14
    Products under Development by Companies, H2 2016 15
    Products under Investigation by Universities/Institutes, H2 2016 16
    Spinocerebellar Ataxia (SCA) - Pipeline by Bio Blast Pharma Ltd, H2 2016 17
    Spinocerebellar Ataxia (SCA) - Pipeline by Biohaven Pharmaceutical Holding Company Limited, H2 2016 18
    Spinocerebellar Ataxia (SCA) - Pipeline by Ionis Pharmaceuticals Inc, H2 2016 19
    Spinocerebellar Ataxia (SCA) - Pipeline by Shionogi & Co Ltd, H2 2016 20
    Spinocerebellar Ataxia (SCA) - Pipeline by Shire Plc, H2 2016 21
    Spinocerebellar Ataxia (SCA) - Pipeline by Spark Therapeutics Inc, H2 2016 22
    Spinocerebellar Ataxia (SCA) - Pipeline by Vybion Inc, H2 2016 23
    Assessment by Monotherapy Products, H2 2016 24
    Number of Products by Stage and Target, H2 2016 26
    Number of Products by Stage and Mechanism of Action, H2 2016 28
    Number of Products by Stage and Route of Administration, H2 2016 30
    Number of Products by Stage and Molecule Type, H2 2016 32
    Spinocerebellar Ataxia (SCA) - Dormant Projects, H2 2016 56

    List of Figures

    Number of Products under Development for Spinocerebellar Ataxia (SCA), H2 2016 8
    Number of Products under Development for Spinocerebellar Ataxia (SCA) - Comparative Analysis, H2 2016 9
    Number of Products under Development by Companies, H2 2016 10
    Number of Products under Investigation by Universities/Institutes, H2 2016 11
    Comparative Analysis by Late Stage Development, H2 2016 12
    Comparative Analysis by Clinical Stage Development, H2 2016 13
    Comparative Analysis by Early Stage Products, H2 2016 14
    Assessment by Monotherapy Products, H2 2016 24
    Number of Products by Targets, H2 2016 25
    Number of Products by Stage and Targets, H2 2016 25
    Number of Products by Mechanism of Actions, H2 2016 27
    Number of Products by Stage and Mechanism of Actions, H2 2016 27
    Number of Products by Routes of Administration, H2 2016 29
    Number of Products by Stage and Routes of Administration, H2 2016 29
    Number of Products by Molecule Types, H2 2016 31
    Number of Products by Stage and Molecule Types, H2 2016 31
    Bio Blast Pharma Ltd
    Biohaven Pharmaceutical Holding Company Limited
    Ionis Pharmaceuticals Inc
    Shionogi & Co Ltd
    Shire Plc
    Spark Therapeutics Inc
    Vybion Inc

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