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遺傳性共濟失調症:開發平台分析

Friedreich Ataxia - Pipeline Review, H2 2016

出版商 Global Markets Direct 商品編碼 219741
出版日期 內容資訊 英文 95 Pages
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遺傳性共濟失調症:開發平台分析 Friedreich Ataxia - Pipeline Review, H2 2016
出版日期: 2016年09月21日 內容資訊: 英文 95 Pages
簡介

遺傳性共濟失調症是影響神經系統和心臟的遺傳性疾病,由於遺傳性缺陷基因所引起。症狀有運動失調、虛弱、痙攣、感覺障礙、骨骼異常、心臟障礙、糖尿病等。

本報告提供全球治療遺傳性共濟失調症所用的開發中產品之開發情形相關分析,提供您產品開發·上市的最新趨勢,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,最新的產業趨勢等資訊。

目錄

簡介

  • 分析範圍

遺傳性共濟失調症概要

治療藥的開發

  • 遺傳性共濟失調症開發中產品:概要
  • 遺傳性共濟失調症開發中產品:比較分析

遺傳性共濟失調症:各企業開發中的治療藥

遺傳性共濟失調症:大學/研究機關研究中的治療藥

遺傳性共濟失調症:開發中產品的概要

  • 後期階段的產品
  • 臨床實驗階段的產品
  • 初期階段的產品

遺傳性共濟失調症:開發中的產品的一覽(各企業)

按遺傳性共濟失調症:研究中的產品的一覽(大學/研究機關)

遺傳性共濟失調症的開發作的企業

  • AAVLife
  • Bioblast Pharma Ltd.
  • BioMarin Pharmaceutical Inc.
  • Biovista Inc.
  • Catabasis Pharmaceuticals, Inc.
  • Edison Pharmaceuticals, Inc.
  • Horizon Pharma Plc
  • MI.TO. Technology S.r.L.
  • Reata Pharmaceuticals, Inc.
  • Retrotope, Inc
  • Shire Plc
  • STATegics, Inc.
  • Varinel, Inc.

遺傳性共濟失調症:治療藥的評估

  • 單獨療法的情況
  • 標的別
  • 各作用機制
  • 各投藥法
  • 各分子類型

藥物簡介

  • BB-FA
    • 產品概要
    • 功能機制
    • 研究開發(R&D)的發展情形
  • BVA-202
  • BVA-203
  • CAT-4001
  • Cell Therapy for Friedreich's Ataxia
  • diazoxide
  • Gene Therapy for Friedreich's ataxia
  • Ataxia
  • interferon gamma-1b
  • Ataxia
  • omaveloxolone
  • RG-2833
  • RG-3250
  • RT-001
  • SHP-622
  • STSE-15
  • VAR-10100
  • VAR-10300
  • vatiquinone

遺傳性共濟失調症:開發中產品的最新趨勢

遺傳性共濟失調症:開發暫停的產品

遺傳性共濟失調症:開發中止的產品

遺傳性共濟失調症相關產品的開發的里程碑

  • 值得注意的最新趨勢·新聞稿

附錄

圖表一覽

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目錄
Product Code: GMDHC8477IDB

Summary

Global Markets Direct's, 'Friedreich Ataxia - Pipeline Review, H2 2016', provides an overview of the Friedreich Ataxia pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Friedreich Ataxia, 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 Friedreich Ataxia 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 Friedreich Ataxia
  • The report reviews pipeline therapeutics for Friedreich Ataxia 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 Friedreich Ataxia therapeutics and enlists all their major and minor projects
  • The report assesses Friedreich Ataxia 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 Friedreich Ataxia

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 Friedreich Ataxia
  • 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 Friedreich Ataxia 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
    • List of Tables
    • List of Figures
  • Introduction
    • Global Markets Direct Report Coverage
  • Friedreich Ataxia Overview
  • Therapeutics Development
    • Pipeline Products for Friedreich Ataxia - Overview
    • Pipeline Products for Friedreich Ataxia - Comparative Analysis
  • Friedreich Ataxia - Therapeutics under Development by Companies
  • Friedreich Ataxia - Therapeutics under Investigation by Universities/Institutes
  • Friedreich Ataxia - Pipeline Products Glance
    • Late Stage Products
    • Clinical Stage Products
    • Early Stage Products
  • Friedreich Ataxia - Products under Development by Companies
  • Friedreich Ataxia - Products under Investigation by Universities/Institutes
  • Friedreich Ataxia - Companies Involved in Therapeutics Development
    • Adverum Biotechnologies, Inc.
    • Almirall, S.A.
    • BioMarin Pharmaceutical Inc.
    • Biovista Inc.
    • Catabasis Pharmaceuticals, Inc.
    • Edison Pharmaceuticals, Inc.
    • Horizon Pharma Plc
    • Ixchel Pharma, LLC
    • ProQR Therapeutics N.V.
    • RaNA Therapeutics, Inc.
    • Reata Pharmaceuticals, Inc.
    • Retrotope, Inc
    • Shire Plc
    • STATegics, Inc.
    • Voyager Therapeutics, Inc.
  • Friedreich Ataxia - Therapeutics Assessment
    • Assessment by Monotherapy Products
    • Assessment by Target
    • Assessment by Mechanism of Action
    • Assessment by Route of Administration
    • Assessment by Molecule Type
  • Drug Profiles
    • AGIL-FA - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • ANN-003 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • BVA-202 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • BVA-203 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • CAT-4001 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • dimethyl fumarate - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Gene Therapy to Activate Frataxin for Friedreich's Ataxia - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Gene Therapy to Activate Frataxin for Friedreich's Ataxia - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Gene Therapy to Activate Frataxin for Friedrich's Ataxia - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • interferon gamma-1b - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • interferon gamma-1b - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • IXC-101 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • JOT-101 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Oligonucleotide to Activate Frataxin for Friedreich's Ataxia - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • omaveloxolone - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • QRX-604 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Recombinant Protein for Friedreich Ataxia - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • RG-3250 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • RT-001 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • RT-002 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • SHP-622 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • STSE-15 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • TAT-FXN - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • vatiquinone - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • VYFXN-01 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
  • Friedreich Ataxia - Dormant Projects
  • Friedreich Ataxia - Discontinued Products
  • Friedreich Ataxia - Product Development Milestones
    • Featured News & Press Releases
      • Aug 02, 2016: Agilis Biotherapeutics Announces FDA Orphan Drug Designation for the Treatment of Friedreich's Ataxia
      • May 05, 2016: Horizon Pharma Completes Target Enrollment of 90 Patients for Phase 3 Trial of ACTIMMUNE(R) (interferon gamma-1b) for the Treatment of People With Friedreich Ataxia
      • Jan 19, 2016: FARA Announces Catabasis Pharmaceuticals as the Recipient of the Kyle Bryant Translational Research Award to Evaluate CAT-4001 as a Potential Therapy for Friedreich's Ataxia
      • Aug 05, 2015: Key Patent Granted For AAVLife's Gene-Therapy Program to Treat Cardiomyopathy in Friedreich's Ataxia
      • Jun 05, 2015: Horizon Pharma Initiates Phase 3 Trial of ACTIMMUNE for the Treatment of People With Friedreich's Ataxia
      • Apr 10, 2015: Horizon Pharma Receives FDA Fast Track Designation for ACTIMMUNE in the Treatment of Friedreich's Ataxia
      • Mar 24, 2015: Catabasis Pharmaceuticals to Present at International Ataxia Research Conference
      • Feb 13, 2015: Horizon Pharma Submits Investigational New Drug Application for ACTIMMUNE in the Treatment of Friedreich's Ataxia
      • Jan 29, 2015: Reata Enrolls First Patient in the MOXIe Study, a Phase 2/3 Study Examining RTA 408 in Friedreich's Ataxia Patients
      • Oct 09, 2014: Horizon Pharma Announces Presentation of ACTIMMUNE Phase 2 Data in Friedreich's Ataxia
      • Oct 03, 2014: Horizon Pharma Receives Orphan-Drug Designation for ACTIMMUNE (Interferon gamma-1b) in Friedreich's Ataxia
      • Sep 30, 2014: Reata Announces the Initiation of Phase 2 Studies Examining RTA 408 for the Treatment of Friedreich's Ataxia and Mitochondrial Myopathies
      • Mar 17, 2014: FDA Awards Fast Track Status to Edison Pharmaceuticals' EPI-743 for Friedreich's Ataxia
      • Feb 04, 2014: FDA Grants Edison Pharmaceuticals' EPI-743 Orphan Status for Friedreich's Ataxia
      • Nov 04, 2013: Edison Pharmaceuticals, FARA, and USF Announce Initiation of EPI-743 Phase 2 Friedreich's Ataxia Clinical Trial in Adults with Point Mutations
  • Appendix
    • Methodology
    • Coverage
    • Secondary Research
    • Primary Research
    • Expert Panel Validation
    • Contact Us
    • Disclaimer

List of Tables

  • Number of Products under Development for Friedreich Ataxia, H2 2016
  • Number of Products under Development for Friedreich Ataxia - Comparative Analysis, H2 2016
  • Number of Products under Development by Companies, H2 2016
  • Number of Products under Investigation by Universities/Institutes, H2 2016
  • Comparative Analysis by Late Stage Development, H2 2016
  • Comparative Analysis by Clinical Stage Development, H2 2016
  • Comparative Analysis by Early Stage Development, H2 2016
  • Products under Development by Companies, H2 2016
  • Products under Investigation by Universities/Institutes, H2 2016
  • Friedreich Ataxia - Pipeline by Adverum Biotechnologies, Inc., H2 2016
  • Friedreich Ataxia - Pipeline by Almirall, S.A., H2 2016
  • Friedreich Ataxia - Pipeline by BioMarin Pharmaceutical Inc., H2 2016
  • Friedreich Ataxia - Pipeline by Biovista Inc., H2 2016
  • Friedreich Ataxia - Pipeline by Catabasis Pharmaceuticals, Inc., H2 2016
  • Friedreich Ataxia - Pipeline by Edison Pharmaceuticals, Inc., H2 2016
  • Friedreich Ataxia - Pipeline by Horizon Pharma Plc, H2 2016
  • Friedreich Ataxia - Pipeline by Ixchel Pharma, LLC, H2 2016
  • Friedreich Ataxia - Pipeline by ProQR Therapeutics N.V., H2 2016
  • Friedreich Ataxia - Pipeline by RaNA Therapeutics, Inc., H2 2016
  • Friedreich Ataxia - Pipeline by Reata Pharmaceuticals, Inc., H2 2016
  • Friedreich Ataxia - Pipeline by Retrotope, Inc, H2 2016
  • Friedreich Ataxia - Pipeline by Shire Plc, H2 2016
  • Friedreich Ataxia - Pipeline by STATegics, Inc., H2 2016
  • Friedreich Ataxia - Pipeline by Voyager Therapeutics, Inc., H2 2016
  • Assessment by Monotherapy Products, H2 2016
  • Number of Products by Stage and Target, H2 2016
  • Number of Products by Stage and Mechanism of Action, H2 2016
  • Number of Products by Stage and Route of Administration, H2 2016
  • Number of Products by Stage and Molecule Type, H2 2016
  • Friedreich Ataxia - Dormant Projects, H2 2016
  • Friedreich Ataxia - Discontinued Products, H2 2016

List of Figures

  • Number of Products under Development for Friedreich Ataxia, H2 2016
  • Number of Products under Development for Friedreich Ataxia - Comparative Analysis, H2 2016
  • Number of Products under Development by Companies, H2 2016
  • Number of Products under Investigation by Universities/Institutes, H2 2016
  • Comparative Analysis by Late Stage Development, H2 2016
  • Comparative Analysis by Clinical Stage Development, H2 2016
  • Comparative Analysis by Early Stage Products, H2 2016
  • Assessment by Monotherapy Products, H2 2016
  • Number of Products by Targets, H2 2016
  • Number of Products by Stage and Targets, H2 2016
  • Number of Products by Top 10 Mechanism of Actions, H2 2016
  • Number of Products by Stage and Top 10 Mechanism of Actions, H2 2016
  • Number of Products by Routes of Administration, H2 2016
  • Number of Products by Stage and Routes of Administration, H2 2016
  • Number of Products by Molecule Types, H2 2016
  • Number of Products by Stage and Molecule Types, H2 2016
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