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脊髓性小腦萎縮症:開發中產品分析

Spinocerebellar Ataxia (SCA) - Pipeline Review, H1 2017

出版商 Global Markets Direct 商品編碼 245961
出版日期 內容資訊 英文 60 Pages
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脊髓性小腦萎縮症:開發中產品分析 Spinocerebellar Ataxia (SCA) - Pipeline Review, H1 2017
出版日期: 2017年05月23日 內容資訊: 英文 60 Pages
簡介

脊髓性小腦萎縮症(SCA)是遺傳性疾病的一種,作用於腦跟脊髓,並引起漸進式的協調障礙(運動失調)。常見的症狀有運動失調,步行不穩,口吃,語言變化,吞嚥障礙等。

本報告提供全球各國治療脊髓性小腦萎縮症所用的開發中產品開發情形相關分析,提供開發中產品的最新趨勢,及臨床實驗的各階段產品一覽,主要企業簡介,主要藥物概要(產品概要,功能機制,研究開發(R&D)的發展情形),最新的產業趨勢等調查結果。

簡介

  • 分析範圍

脊髓性小腦萎縮症概要

治療藥的開發

  • 脊髓性小腦萎縮症轉動開發中產品:概要
  • 各企業的開發平台
  • 大學/研究機關所開發的開發平台
  • 企業開發中的產品
  • 大學/研究機關開發中的產品

治療藥的評估

  • 標的別
  • 各作用機制
  • 各給藥途徑
  • 各分子類型

脊髓性小腦萎縮症開發治療藥的企業

  • Bio Blast Pharma Ltd
  • Ionis Pharmaceuticals Inc
  • Reata Pharmaceuticals Inc
  • 鹽野義製藥
  • Shire Plc
  • Spark Therapeutics Inc
  • Vybion Inc

藥物簡介

開發暫停的產品

產品開發的里程碑

  • 最新消息及新聞稿

附錄

圖表一覽

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目錄
Product Code: GMDHC9318IDB

Summary

Global Markets Direct'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

Global Markets Direct'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 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.

Table of Contents

  • Table of Contents
    • List of Tables
    • List of Figures
  • Introduction
    • Global Markets Direct 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
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