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溶小體儲積症 : 開發平台分析

Lysosomal Storage Disorder - Pipeline Review, H2 2015

出版商 Global Markets Direct 商品編碼 213089
出版日期 內容資訊 英文 78 Pages
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溶小體儲積症 : 開發平台分析 Lysosomal Storage Disorder - Pipeline Review, H2 2015
出版日期: 2015年09月23日 內容資訊: 英文 78 Pages
簡介

溶小體儲積症(LSD)是各種先天性罕見疾病中的一大類,是體內儲積未消化(或是並未完全消化)的高分子,造成細胞機能障礙或臨床上的異常。主要症狀有重覆感染及疝氣、發育遲緩、骨骼疼痛和心臟障礙等。主要的治療方法有基質合成抑制療法(SSI)和酵素替代療法(ERT),幹細胞移植等。

本報告提供全球各國治療溶小體儲積症(LSD)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

溶小體儲積症概要

治療藥的開發

  • 溶小體儲積症開發中產品:概要
  • 溶小體儲積症開發中產品:比較分析

各企業開發中的溶小體儲積症治療藥

大學/研究機關研究中的溶小體儲積症治療藥

開發中產品的概要

  • 臨床實驗階段的產品
  • 初期階段的產品
  • 開發階段不明的產品

溶小體儲積症治療藥:開發中的產品的一覽(各企業)

溶小體儲積症治療藥:研究中的產品的一覽(大學/研究機關別)

溶小體儲積症治療藥的開發企業

  • 2-BBB Medicines BV
  • Amicus Therapeutics, Inc.
  • AngioChem Inc.
  • Fate Therapeutics, Inc.
  • Minoryx Therapeutics s.l.
  • Nimbus Therapeutics, LLC
  • Nuo Therapeutics, Inc.
  • Orphazyme ApS
  • Oxyrane Belgium NV
  • Promosome, LLC
  • REGiMMUNE Corporation
  • Sangamo BioSciences, Inc.
  • Ultragenyx Pharmaceutical Inc.

溶小體儲積症:治療藥的評估

  • 單劑治療藥的情況
  • 標的別
  • 各作用機制
  • 各投藥法
  • 各分子類型

藥物簡介

  • 2B3-201
    • 產品概要
    • 功能機制
    • 研究開發(R&D)的發展情形
  • ALD-601
  • Chaperone-ERT Combinations
  • Delta-tocopherol
  • FT-1050
  • ML-SA1
  • OR-02
  • OR-03
  • Recombinant Enzyme for Farber Disease and Cystic Fibrosis
  • Recombinant Enzyme for LSD-2 Disease
  • Recombinant Enzyme for LSD-3 Disease
  • Recombinant Enzyme for LSD-4 Disease
  • Recombinant Enzyme for LSD-5 Disease
  • Recombinant Enzyme for Lysosomal Storage Diseases and Metabolic Disorders
  • Recombinant Enzyme for Lysosomal Storage Disorder
  • Recombinant Enzyme to Target LRP1 for Lysosomal Storage Disorder
  • RGI-5000
  • SBLSD-3
  • SBLSD-4
  • Small Molecule to Activate Beta-Galactosidase for GM1 Gangliosidosis and Gaucher's disease
  • Small Molecules for GM1-gangliosidosis and Morquio B
  • Small Molecules for Lysosomal Storage Disorders
  • Stem Cell Therapy for Lysosomal Storage Disorder
  • UX-004

溶小體儲積症治療藥:開發中產品的最新趨勢

溶小體儲積症治療藥:暫停開發的產品

溶小體儲積症相關產品的開發里程碑

  • 值得注意的最新趨勢·新聞稿
  • ■附錄

圖表一覽

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: GMDHC7092IDB

Summary

Global Markets Direct's, 'Lysosomal Storage Disorder - Pipeline Review, H2 2015', provides an overview of the Lysosomal Storage Disorder's therapeutic pipeline.

This report provides comprehensive information on the therapeutic development for Lysosomal Storage Disorder, complete with comparative analysis at various stages, therapeutics assessment by drug target, mechanism of action (MoA), route of administration (RoA) and molecule type, along with latest updates, and featured news and press releases. It also reviews key players involved in the therapeutic development for Lysosomal Storage Disorder and special features on late-stage 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, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources, put together by Global Markets Direct's team. Drug profiles/records featured in the report undergoes periodic updation following a stringent set of processes that ensures that all the profiles are updated with the latest set of information. Additionally, processes including live news & deals tracking, browser based alert-box and clinical trials registries tracking ensure that the most recent developments are captured on a real time basis.

The report enhances decision making capabilities and help to create effective counter strategies to gain competitive advantage. It strengthens R&D pipelines by identifying new targets and MOAs to produce first-in-class and best-in-class products.

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

Scope

  • The report provides a snapshot of the global therapeutic landscape of Lysosomal Storage Disorder
  • The report reviews key pipeline products under drug profile section which includes, product description, MoA and R&D brief, licensing and collaboration details & other developmental activities
  • The report reviews key players involved in the therapeutics development for Lysosomal Storage Disorder and enlists all their major and minor projects
  • The report summarizes all the dormant and discontinued pipeline projects
  • A review of the Lysosomal Storage Disorder products under development by companies and universities/research institutes based on information derived from company and industry-specific sources
  • Pipeline products coverage based on various stages of development ranging from pre-registration till discovery and undisclosed stages
  • A detailed assessment of monotherapy and combination therapy pipeline projects
  • Coverage of the Lysosomal Storage Disorder pipeline on the basis of target, MoA, route of administration and molecule type
  • Latest news and deals relating related to pipeline products

Reasons to buy

  • Provides strategically significant competitor information, analysis, and insights to formulate effective R&D development strategies
  • Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage
  • Develop strategic initiatives by understanding the focus areas of leading companies
  • Identify and understand important and diverse types of therapeutics under development for Lysosomal Storage Disorder
  • Plan mergers and acquisitions effectively by identifying key players of the most promising pipeline
  • Devise corrective measures for pipeline projects by understanding Lysosomal Storage Disorder 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
  • Lysosomal Storage Disorder Overview
  • Therapeutics Development
    • Pipeline Products for Lysosomal Storage Disorder - Overview
    • Pipeline Products for Lysosomal Storage Disorder - Comparative Analysis
  • Lysosomal Storage Disorder - Therapeutics under Development by Companies
  • Lysosomal Storage Disorder - Therapeutics under Investigation by Universities/Institutes
  • Lysosomal Storage Disorder - Pipeline Products Glance
    • Clinical Stage Products
    • Early Stage Products
    • Unknown Stage Products
  • Lysosomal Storage Disorder - Products under Development by Companies
  • Lysosomal Storage Disorder - Products under Investigation by Universities/Institutes
  • Lysosomal Storage Disorder - Companies Involved in Therapeutics Development
    • 2-BBB Medicines BV
    • Amicus Therapeutics, Inc.
    • AngioChem Inc.
    • Fate Therapeutics, Inc.
    • Minoryx Therapeutics s.l.
    • Nimbus Therapeutics, LLC
    • Nuo Therapeutics, Inc.
    • Orphazyme ApS
    • Oxyrane Belgium NV
    • Promosome, LLC
    • REGiMMUNE Corporation
    • Sangamo BioSciences, Inc.
    • Ultragenyx Pharmaceutical Inc.
  • Lysosomal Storage Disorder - 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
    • 2B3-201 - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • ALD-601 - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • Chaperone-ERT Combinations - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • Delta-tocopherol - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • FT-1050 - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • ML-SA1 - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • OR-02 - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • OR-03 - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • Recombinant Enzyme for Farber Disease and Cystic Fibrosis - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • Recombinant Enzyme for LSD-2 Disease - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • Recombinant Enzyme for LSD-3 Disease - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • Recombinant Enzyme for LSD-4 Disease - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • Recombinant Enzyme for LSD-5 Disease - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • Recombinant Enzyme for Lysosomal Storage Diseases and Metabolic Disorders - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • Recombinant Enzyme for Lysosomal Storage Disorder - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • Recombinant Enzyme to Target LRP1 for Lysosomal Storage Disorder - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • RGI-5000 - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • SBLSD-3 - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • SBLSD-4 - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • Small Molecule to Activate Beta-Galactosidase for GM1 Gangliosidosis and Gaucher's disease - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • Small Molecules for GM1-gangliosidosis and Morquio B - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • Small Molecules for Lysosomal Storage Disorders - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • Stem Cell Therapy for Lysosomal Storage Disorder - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
    • UX-004 - Drug Profile
      • Product Description
      • Mechanism of Action
      • R&D Progress
  • Lysosomal Storage Disorder - Recent Pipeline Updates
  • Lysosomal Storage Disorder - Dormant Projects
  • Lysosomal Storage Disorder - Product Development Milestones
    • Featured News & Press Releases
      • Jul 29, 2014: Fate Therapeutics Announces FDA Clearance of IND for Clinical Development of PROHEMA in Inherited Metabolic Disorders
      • Feb 26, 2014: Fate Therapeutics Announces Observed Effects of Pharmacologic Modulation on T Cell Compartment From Its Phase 1b Study of PROHEMA
      • Oct 21, 2013: For first time, drug developed based on zebrafish studies passes Phase I clinical trial
      • Aug 15, 2012: Orphazyme Receives European Patent Covering Use Of HSP70 For Treatment Of Lysosomal Storage Diseases
  • Appendix
    • Methodology
    • Coverage
    • Secondary Research
    • Primary Research
    • Expert Panel Validation
    • Contact Us
    • Disclaimer

List of Tables

  • Number of Products under Development for Lysosomal Storage Disorder, H2 2015
  • Number of Products under Development for Lysosomal Storage Disorder - Comparative Analysis, H2 2015
  • Number of Products under Development by Companies, H2 2015
  • Number of Products under Investigation by Universities/Institutes, H2 2015
  • Comparative Analysis by Clinical Stage Development, H2 2015
  • Comparative Analysis by Early Stage Development, H2 2015
  • Comparative Analysis by Unknown Stage Development, H2 2015
  • Products under Development by Companies, H2 2015
  • Products under Development by Companies, H2 2015 (Contd..1)
  • Products under Investigation by Universities/Institutes, H2 2015
  • Lysosomal Storage Disorder - Pipeline by 2-BBB Medicines BV, H2 2015
  • Lysosomal Storage Disorder - Pipeline by Amicus Therapeutics, Inc., H2 2015
  • Lysosomal Storage Disorder - Pipeline by AngioChem Inc., H2 2015
  • Lysosomal Storage Disorder - Pipeline by Fate Therapeutics, Inc., H2 2015
  • Lysosomal Storage Disorder - Pipeline by Minoryx Therapeutics s.l., H2 2015
  • Lysosomal Storage Disorder - Pipeline by Nimbus Therapeutics, LLC, H2 2015
  • Lysosomal Storage Disorder - Pipeline by Nuo Therapeutics, Inc., H2 2015
  • Lysosomal Storage Disorder - Pipeline by Orphazyme ApS, H2 2015
  • Lysosomal Storage Disorder - Pipeline by Oxyrane Belgium NV, H2 2015
  • Lysosomal Storage Disorder - Pipeline by Promosome, LLC, H2 2015
  • Lysosomal Storage Disorder - Pipeline by REGiMMUNE Corporation, H2 2015
  • Lysosomal Storage Disorder - Pipeline by Sangamo BioSciences, Inc., H2 2015
  • Lysosomal Storage Disorder - Pipeline by Ultragenyx Pharmaceutical Inc., H2 2015
  • Assessment by Monotherapy Products, H2 2015
  • Number of Products by Stage and Target, H2 2015
  • Number of Products by Stage and Mechanism of Action, H2 2015
  • Number of Products by Stage and Route of Administration, H2 2015
  • Number of Products by Stage and Molecule Type, H2 2015
  • Lysosomal Storage Disorder Therapeutics - Recent Pipeline Updates, H2 2015
  • Lysosomal Storage Disorder - Dormant Projects, H2 2015

List of Figures

  • Number of Products under Development for Lysosomal Storage Disorder, H2 2015
  • Number of Products under Development for Lysosomal Storage Disorder - Comparative Analysis, H2 2015
  • Number of Products under Development by Companies, H2 2015
  • Number of Products under Investigation by Universities/Institutes, H2 2015
  • Comparative Analysis by Early Stage Products, H2 2015
  • Assessment by Monotherapy Products, H2 2015
  • Number of Products by Top 10 Targets, H2 2015
  • Number of Products by Stage and Top 10 Targets, H2 2015
  • Number of Products by Top 10 Mechanism of Actions, H2 2015
  • Number of Products by Stage and Top 10 Mechanism of Actions, H2 2015
  • Number of Products by Top 10 Routes of Administration, H2 2015
  • Number of Products by Stage and Top 10 Routes of Administration, H2 2015
  • Number of Products by Top 10 Molecule Types, H2 2015
  • Number of Products by Stage and Top 10 Molecule Types, H2 2015
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