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黏多醣症I(MPS I,賀勒氏症):開發平台分析

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline Review, H1 2016

出版商 Global Markets Direct 商品編碼 245985
出版日期 內容資訊 英文 87 Pages
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黏多醣症I(MPS I,賀勒氏症):開發平台分析 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline Review, H1 2016
出版日期: 2016年06月08日 內容資訊: 英文 87 Pages
簡介

賀勒氏症是罕見的難治疾病之一,是指人體內無法分解被稱為糖胺聚糖(舊稱:黏多醣體)的多糖(糖分子長連鎖)這樣的遺傳性代謝疾病。賀勒氏症屬於黏多醣症(MPS)的疾病群組。賀勒氏症患者由於無法生產α-L-iduronidase(溶酶體酶的一種)。該酵素的作用是支援糖胺聚糖的分解。這個分子常見於人體各部位、尤其是關節週圍的黏液及體液。其症狀有脊椎骨異常,爪形手,角膜混濁,聽障,心臟瓣膜症等。治療方法有骨髓移植,酵素療法,基因治療等。

本報告提供全球各國的黏多醣症I(MPS I,賀勒氏症)所用的開發中產品之開發情形相關分析,提供您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。

簡介

  • 分析範圍

黏多醣症I概要

治療藥的開發

  • 黏多醣症I開發中產品:概要
  • 黏多醣症I開發中產品:比較分析

各企業正在開發的黏多醣症I治療藥

各大學·研究機關研究中的黏多醣症I治療藥

開發中產品的概要

  • 臨床階段的產品
  • 初期階段的產品
  • 開發階段不明的產品

黏多醣症I開發治療藥的企業

  • AngioChem Inc.
  • ArmaGen Inc.
  • Athersys, Inc.
  • Bioasis Technologies Inc.
  • Eloxx Pharmaceuticals, Ltd.
  • Inventiva
  • OPKO Health, Inc.
  • PTC Therapeutics, Inc.
  • RegenxBio Inc.
  • Sangamo BioSciences, Inc.

黏多醣症I:治療藥的評估

  • 單劑治療藥的情況
  • 標的別
  • 各行動機制
  • 各投藥法
  • 各分子類型

藥物簡介

黏多醣症I治療藥:開發暫停的產品

黏多醣症I治療藥:開發中止的產品

黏多醣症I相關產品的開發的里程碑

  • 最新消息及新聞稿

附錄

圖表

本網頁內容可能與最新版本有所差異。詳細情況請與我們聯繫。

目錄
Product Code: GMDHC8194IDB

Summary

Global Markets Direct's, 'Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline Review, H1 2016', provides an overview of the Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ), 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 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) 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 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome )
  • The report reviews pipeline therapeutics for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) 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 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) therapeutics and enlists all their major and minor projects
  • The report assesses Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) 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 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome )

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 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome )
  • 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 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) 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
  • Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) Overview
  • Therapeutics Development
    • Pipeline Products for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Overview
    • Pipeline Products for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Comparative Analysis
  • Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Therapeutics under Development by Companies
  • Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Therapeutics under Investigation by Universities/Institutes
  • Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline Products Glance
    • Clinical Stage Products
    • Early Stage Products
    • Unknown Stage Products
  • Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Products under Development by Companies
  • Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Products under Investigation by Universities/Institutes
  • Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Companies Involved in Therapeutics Development
    • AngioChem Inc.
    • ArmaGen Inc.
    • Athersys, Inc.
    • Bioasis Technologies Inc.
    • Eloxx Pharmaceuticals, Ltd.
    • Inventiva
    • OPKO Health, Inc.
    • PTC Therapeutics, Inc.
    • RegenxBio Inc.
    • Sangamo BioSciences, Inc.
  • Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - 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
    • AGT-181 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • ataluren - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Cell Therapy for Mucopolysaccharidosis I - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Cell Therapy to Activate Iduronidase for Hurler Syndrome - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • odiparcil - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Oligonucleotide for Mucopolysaccharidosis I - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • pentosan polysulfate sodium - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • PF-05285401 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Recombinant Alpha-L-Iduronidase Replacement for Hurler Syndrome - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Recombinant Alpha-L-Iduronidase Replacement for Mucopolysaccharidosis I - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Recombinant Alpha-L-Iduronidase Replacement for Mucopolysaccharidosis I - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • RGX-111 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • SB-318 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Small Molecules for Hurler Syndrome - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Stem Cell Therapy for Type1 Mucopolysaccharidosis - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
  • Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Dormant Projects
  • Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Discontinued Products
  • Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Product Development Milestones
    • Featured News & Press Releases
      • May 09, 2016: Sangamo BioSciences Presents Recent Developments From Research And ZFP Therapeutic Programs In Multiple Presentations At Annual Meeting of the American Society of Gene and Cell Therapy
      • Mar 31, 2016: ArmaGen Announces Initiation of Phase 2 Proof-of-Concept Clinical Trial in Brazil to Study AGT-181 for the Treatment of Hurler Syndrome
      • Feb 08, 2016: Sangamo BioSciences Announces FDA Clearance Of Investigational New Drug Application For ZFN-Mediated Genome Editing Treatment Of MPS I
      • Dec 30, 2015: FDA Grants Rare Pediatric Disease Designation to REGENXBIO RGX-111 Gene Therapy for the Treatment of Mucopolysaccharidosis Type I (MPS I)
      • Nov 05, 2015: ArmaGen Receives Rare Pediatric Disease Designation from FDA for AGT-181 for the Potential Treatment of Hurler Syndrome
      • Oct 01, 2015: FDA Grants Orphan Drug Designation to REGENXBIO's RGX-111 Gene Therapy for the Treatment of Mucopolysaccharidosis Type I
      • Sep 02, 2015: ArmaGen Announces First Patient Dosed in Phase 1/2a Clinical Trial of AGT-181 for the Treatment of Hurler Syndrome
      • Apr 08, 2015: ArmaGen Announces FDA Acceptance of IND Application for AGT-181 for the Treatment of Hurler Syndrome
      • Dec 19, 2014: Translarna Granted Orphan Drug Designation in the U.S. and Europe for the Treatment of Mucopolysaccharidosis I
      • Feb 05, 2014: Mouse Study Shows Gene Therapy May Be Possible Cure for Hurler Syndrome
      • Jul 10, 2012: Athersys Receives Orphan Drug Designation For MultiStem In Hurler's Syndrome
      • Apr 20, 2012: Athersys Announces Publication Of Results Of Preclinical Study Of MultiStem In Cell Transplant
  • Appendix
    • Methodology
    • Coverage
    • Secondary Research
    • Primary Research
    • Expert Panel Validation
    • Contact Us
    • Disclaimer

List of Tables

  • Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ), H1 2016
  • Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Comparative Analysis, H1 2016
  • Number of Products under Development by Companies, H1 2016
  • Number of Products under Investigation by Universities/Institutes, H1 2016
  • Comparative Analysis by Clinical Stage Development, H1 2016
  • Comparative Analysis by Early Stage Development, H1 2016
  • Comparative Analysis by Unknown Stage Development, H1 2016
  • Products under Development by Companies, H1 2016
  • Products under Investigation by Universities/Institutes, H1 2016
  • Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by AngioChem Inc., H1 2016
  • Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by ArmaGen Inc., H1 2016
  • Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Athersys, Inc., H1 2016
  • Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Bioasis Technologies Inc., H1 2016
  • Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Eloxx Pharmaceuticals, Ltd., H1 2016
  • Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Inventiva, H1 2016
  • Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by OPKO Health, Inc., H1 2016
  • Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by PTC Therapeutics, Inc., H1 2016
  • Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by RegenxBio Inc., H1 2016
  • Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Sangamo BioSciences, Inc., H1 2016
  • Assessment by Monotherapy Products, H1 2016
  • Number of Products by Stage and Target, H1 2016
  • Number of Products by Stage and Mechanism of Action, H1 2016
  • Number of Products by Stage and Route of Administration, H1 2016
  • Number of Products by Stage and Molecule Type, H1 2016
  • Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Dormant Projects, H1 2016
  • Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Discontinued Products, H1 2016

List of Figures

  • Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ), H1 2016
  • Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Comparative Analysis, H1 2016
  • Number of Products under Development by Companies, H1 2016
  • Comparative Analysis by Early Stage Products, H1 2016
  • Assessment by Monotherapy Products, H1 2016
  • Number of Products by Targets, H1 2016
  • Number of Products by Stage and Targets, H1 2016
  • Number of Products by Mechanism of Actions, H1 2016
  • Number of Products by Stage and Mechanism of Actions, H1 2016
  • Number of Products by Routes of Administration, H1 2016
  • Number of Products by Stage and Routes of Administration, H1 2016
  • Number of Products by Molecule Types, H1 2016
  • Number of Products by Stage and Molecule Types, H1 2016
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