Summary
Global Markets Direct'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
Global Markets Direct'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 Global Markets Direct’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.
Companies Mentioned
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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Table of Contents
Table of Contents 2
List of Tables 5
List of Figures 5
Introduction 6
Global Markets Direct 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
