Spinocerebellar Ataxia (SCA) - Pipeline Review, H1 2017

  • ID: 4267362
  • Report
  • 60 pages
  • Global Markets Direct
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FEATURED COMPANIES

  • Bio Blast Pharma Ltd
  • Ionis Pharmaceuticals Inc
  • Reata Pharmaceuticals Inc
  • Shionogi & Co Ltd
  • Shire Plc
  • Spark Therapeutics Inc
  • MORE
Spinocerebellar Ataxia (SCA) - Pipeline Review, H1 2017

Summary:

The 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:

The latest 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 our 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.
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Note: Product cover images may vary from those shown
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FEATURED COMPANIES

  • Bio Blast Pharma Ltd
  • Ionis Pharmaceuticals Inc
  • Reata Pharmaceuticals Inc
  • Shionogi & Co Ltd
  • Shire Plc
  • Spark Therapeutics Inc
  • MORE
Introduction

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

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

Nov 19, 2014: Bio Blast Pharma Receives Orphan Drug Designation From U.S. FDA for Cabaletta for the Treatment of Spinocerebellar Ataxia Type

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

Number of Products under Development by Companies, H1

Number of Products under Development by Universities/Institutes, H1

Products under Development by Companies, H1

Products under Development by Universities/Institutes, H1

Number of Products by Stage and Target, H1

Number of Products by Stage and Mechanism of Action, H1

Number of Products by Stage and Route of Administration, H1

Number of Products by Stage and Molecule Type, H1

Spinocerebellar Ataxia (SCA) - Pipeline by Bio Blast Pharma Ltd, H1

Spinocerebellar Ataxia (SCA) - Pipeline by Ionis Pharmaceuticals Inc, H1

Spinocerebellar Ataxia (SCA) - Pipeline by Reata Pharmaceuticals Inc, H1

Spinocerebellar Ataxia (SCA) - Pipeline by Shionogi & Co Ltd, H1

Spinocerebellar Ataxia (SCA) - Pipeline by Shire Plc, H1

Spinocerebellar Ataxia (SCA) - Pipeline by Spark Therapeutics Inc, H1

Spinocerebellar Ataxia (SCA) - Pipeline by Vybion Inc, H1

Spinocerebellar Ataxia (SCA) - Dormant Projects, H1

List of Figures:

Number of Products under Development for Spinocerebellar Ataxia (SCA), H1

Number of Products under Development by Companies, H1

Number of Products under Development by Universities/Institutes, H1

Number of Products by Targets, H1

Number of Products by Stage and Targets, H1

Number of Products by Top 10 Mechanism of Actions, H1

Number of Products by Stage and Top 10 Mechanism of Actions, H1

Number of Products by Routes of Administration, H1

Number of Products by Stage and Routes of Administration, H1

Number of Products by Molecule Types, H1

Number of Products by Stage and Molecule Types, H
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  • Bio Blast Pharma Ltd
  • Ionis Pharmaceuticals Inc
  • Reata Pharmaceuticals Inc
  • Shionogi & Co Ltd
  • Shire Plc
  • Spark Therapeutics Inc
  • Vybion Inc
Note: Product cover images may vary from those shown
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Note: Product cover images may vary from those shown
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