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Spinocerebellar Ataxia (SCA) - Pipeline Review, H1 2016

Published: May 31, 2016 | Pages: 55 | Publisher: Global Markets Direct | Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

Spinocerebellar Ataxia (SCA) - Pipeline Review, H1 2016

Summary

Global Markets Direct's, 'Spinocerebellar Ataxia (SCA) - Pipeline Review, H1 2016', provides an overview of the Spinocerebellar Ataxia (SCA) pipeline landscape. 

The report provides comprehensive information on the therapeutics under development for Spinocerebellar Ataxia (SCA), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Spinocerebellar Ataxia (SCA) and features dormant and discontinued projects. 

Global Markets Direct's report features investigational drugs from across globe covering over 20 therapy areas and nearly 3,000 indications. The report 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. Drug profiles featured in the report undergoes periodic review following a stringent set of processes to ensure that all the profiles are updated with the latest set of information. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

The report 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.

Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data.

Scope

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


Reasons To Buy

- Gain strategically significant competitor information, analysis, and insights to formulate effective R&D strategies
- Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage 
- Identify and understand important and diverse types of therapeutics under development for Spinocerebellar Ataxia (SCA)
- Identify potential new clients or partners in the target demographic
- Develop strategic initiatives by understanding the focus areas of leading companies 
- Plan mergers and acquisitions effectively by identifying key players and it's most promising pipeline therapeutics
- Devise corrective measures for pipeline projects by understanding Spinocerebellar Ataxia (SCA) 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
- Modify the therapeutic portfolio by identifying discontinued projects and understanding the factors that drove them from pipeline
 Table of Contents
Table of Contents 2 List of Tables 4 List of Figures 4 Introduction 5 Global Markets Direct Report Coverage 5 Spinocerebellar Ataxia (SCA) Overview 6 Therapeutics Development 7 Pipeline Products for Spinocerebellar Ataxia (SCA) - Overview 7 Pipeline Products for Spinocerebellar Ataxia (SCA) - Comparative Analysis 8 Spinocerebellar Ataxia (SCA) - Therapeutics under Development by Companies 9 Spinocerebellar Ataxia (SCA) - Therapeutics under Investigation by Universities/Institutes 10 Spinocerebellar Ataxia (SCA) - Pipeline Products Glance 11 Late Stage Products 11 Clinical Stage Products 12 Early Stage Products 13 Spinocerebellar Ataxia (SCA) - Products under Development by Companies 14 Spinocerebellar Ataxia (SCA) - Products under Investigation by Universities/Institutes 15 Spinocerebellar Ataxia (SCA) - Companies Involved in Therapeutics Development 16 Baxalta Incorporated 16 Bioblast Pharma Ltd. 17 Kissei Pharmaceutical Co., Ltd. 18 Vybion, Inc. 19 Spinocerebellar Ataxia (SCA) - Therapeutics Assessment 20 Assessment by Monotherapy Products 20 Assessment by Target 21 Assessment by Mechanism of Action 23 Assessment by Route of Administration 25 Assessment by Molecule Type 27 Drug Profiles 29 Antisense RNAi Oligonucleotide to Inhibit Ataxin-7 for Spinocerebellar Ataxia Type 7 - Drug Profile 29 Product Description 29 Mechanism of Action 29 R&D Progress 29 epoetin alfa - Drug Profile 30 Product Description 30 Mechanism of Action 30 R&D Progress 30 immune globulin (human) - Drug Profile 31 Product Description 31 Mechanism of Action 31 R&D Progress 31 INT-41 - Drug Profile 34 Product Description 34 Mechanism of Action 34 R&D Progress 34 riluzole - Drug Profile 36 Product Description 36 Mechanism of Action 36 R&D Progress 36 RNAi Gene Therapy for Machado-Joseph Disease - Drug Profile 38 Product Description 38 Mechanism of Action 38 R&D Progress 38 rovatirelin - Drug Profile 39 Product Description 39 Mechanism of Action 39 R&D Progress 39 Stem Cell Therapy for Osteoarthritis, Diabetes Mellitus, Acute Liver Failure and Neurodegenerative Diseases - Drug Profile 40 Product Description 40 Mechanism of Action 40 R&D Progress 40 trehalose - Drug Profile 42 Product Description 42 Mechanism of Action 42 R&D Progress 42 Spinocerebellar Ataxia (SCA) - Recent Pipeline Updates 43 Spinocerebellar Ataxia (SCA) - Dormant Projects 50 Spinocerebellar Ataxia (SCA) - Product Development Milestones 51 Featured News & Press Releases 51 Mar 03, 2016: Portage Biohaven orphan drug designation request granted for the treatment of spinocerebellar ataxia 51 Sep 14, 2015: BioBlast Granted Patent From USPTO for Cabaletta for the Treatment of Spinocerebellar Ataxia 52 Jun 29, 2015: BioBlast Pharma Receives Orphan Drug Designation From the European Commission for Cabaletta for the Treatment of Spinocerebellar Ataxia 52 Mar 30, 2015: BioBlast Pharma Announces Positive in vivo Preclinical Proof-of-Concept Results of Its Cabaletta Platform in Spinocerebellar Ataxia Type 3 52 Nov 19, 2014: Bio Blast Pharma Receives Orphan Drug Designation From U.S. FDA for Cabaletta for the Treatment of Spinocerebellar Ataxia Type 3 53 Appendix 54 Methodology 54 Coverage 54 Secondary Research 54 Primary Research 54 Expert Panel Validation 54 Contact Us 54 Disclaimer 55
List of Tables
Number of Products under Development for Spinocerebellar Ataxia (SCA), H1 2016 7 Number of Products under Development for Spinocerebellar Ataxia (SCA) - Comparative Analysis, H1 2016 8 Number of Products under Development by Companies, H1 2016 9 Number of Products under Investigation by Universities/Institutes, H1 2016 10 Comparative Analysis by Late Stage Development, H1 2016 11 Comparative Analysis by Clinical Stage Development, H1 2016 12 Comparative Analysis by Early Stage Development, H1 2016 13 Products under Development by Companies, H1 2016 14 Products under Investigation by Universities/Institutes, H1 2016 15 Spinocerebellar Ataxia (SCA) - Pipeline by Baxalta Incorporated, H1 2016 16 Spinocerebellar Ataxia (SCA) - Pipeline by Bioblast Pharma Ltd., H1 2016 17 Spinocerebellar Ataxia (SCA) - Pipeline by Kissei Pharmaceutical Co., Ltd., H1 2016 18 Spinocerebellar Ataxia (SCA) - Pipeline by Vybion, Inc., H1 2016 19 Assessment by Monotherapy Products, H1 2016 20 Number of Products by Stage and Target, H1 2016 22 Number of Products by Stage and Mechanism of Action, H1 2016 24 Number of Products by Stage and Route of Administration, H1 2016 26 Number of Products by Stage and Molecule Type, H1 2016 28 Spinocerebellar Ataxia (SCA) Therapeutics - Recent Pipeline Updates, H1 2016 43 Spinocerebellar Ataxia (SCA) - Dormant Projects, H1 2016 50


List of Figures
Number of Products under Development for Spinocerebellar Ataxia (SCA), H1 2016 7 Number of Products under Development for Spinocerebellar Ataxia (SCA) - Comparative Analysis, H1 2016 8 Number of Products under Development by Companies, H1 2016 9 Number of Products under Investigation by Universities/Institutes, H1 2016 10 Comparative Analysis by Clinical Stage Development, H1 2016 12 Comparative Analysis by Early Stage Products, H1 2016 13 Assessment by Monotherapy Products, H1 2016 20 Number of Products by Targets, H1 2016 21 Number of Products by Stage and Targets, H1 2016 21 Number of Products by Mechanism of Actions, H1 2016 23 Number of Products by Stage and Mechanism of Actions, H1 2016 23 Number of Products by Routes of Administration, H1 2016 25 Number of Products by Stage and Routes of Administration, H1 2016 25 Number of Products by Molecule Types, H1 2016 27 Number of Products by Stage and Molecule Types, H1 2016 27

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