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ライソゾーム病 : パイプライン分析

Lysosomal Storage Disorder - Pipeline Review, H2 2015

発行 Global Markets Direct 商品コード 213089
出版日 ページ情報 英文 78 Pages
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ライソゾーム病 : パイプライン分析 Lysosomal Storage Disorder - Pipeline Review, H2 2015
出版日: 2015年09月23日 ページ情報: 英文 78 Pages
概要

ライソゾーム病(LSD、リソソーム蓄積病とも)とは様々な先天性難病の一群で、未消化の(または完全に消化されていない)高分子が蓄積して、採取的には細胞機能障害や臨床上の異常を発症する、というものです。主な症状として、回帰感染やヘルニア、発達遅滞、骨痛や心臓障害などが挙げられます。主な治療法には、基質合成抑制療法(SSI)や酵素置換療法(ERT)、幹細胞移植などがあります。

当レポートでは、世界各国でのライソゾーム病(LSD)治療用のパイプライン製品の開発状況について分析し、製品開発・上市の最新動向や、治験の各段階にある製品の一覧、主要企業のプロファイル、主な薬剤の概要、最新の業界動向などの情報をお届けします。

目次

イントロダクション

  • 分析範囲

ライソゾーム病の概要

治療薬の開発

  • ライソゾーム病向けパイプライン製品:概要
  • ライソゾーム病向けパイプライン製品:比較分析

各企業で開発中のライソゾーム病治療薬

大学/研究機関で研究中のライソゾーム病治療薬

パイプライン製品の概略

  • 治験段階の製品
  • 初期段階の製品
  • 開発段階が不明の製品

ライソゾーム病治療薬:開発中の製品の一覧(企業別)

ライソゾーム病治療薬:研究中の製品の一覧(大学/研究機関別)

ライソゾーム病治療薬の開発に従事している企業

  • 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.

ライソゾーム病:治療薬の評価

  • 単剤治療薬の場合
  • 標的別
  • 作用機序別
  • 投与方法別
  • 分子の種類別

薬剤のプロファイル

  • 2B3-201
    • 製品概要
    • 機能メカニズム
    • 研究開発(R&D)の進展状況
  • ALD-601
  • シャペロンと酵素置換療法(ERT)の併用
  • Delta-tocopherol
  • FT-1050
  • ML-SA1
  • OR-02
  • OR-03
  • ファーバー病および嚢胞性線維症向け遺伝子組み換え型酵素LSD-2疾患向け遺伝子組み換え型酵素
  • LSD-3疾患向け遺伝子組み換え型酵素
  • LSD-4疾患向け遺伝子組み換え型酵素
  • LSD-5疾患向け遺伝子組み換え型酵素
  • ライソゾーム病および代謝性疾患向け遺伝子組み換え型酵素
  • ライソゾーム病向け遺伝子組み換え型酵素
  • ライソゾーム病向けLRP1標的遺伝子組み換え型酵素
  • RGI-5000
  • SBLSD-3
  • SBLSD-4
  • GMIガングリオシドーシスおよびゴーシェ病向けβガラクトシダーゼ活性小分子
  • GM1ガングリオシドおよびモルキオB向け治療法
  • ライソゾーム病向け小分子薬
  • ライソゾーム病向け幹細胞療法
  • UX-004

ライソゾーム病治療薬:パイプライン製品の最新動向

ライソゾーム病治療薬:開発が休止状態の製品

ライソゾーム病関連製品の開発のマイルストーン

  • 注目すべき最新動向・プレスリリース
  • ■付録

図表一覧

このページに掲載されている内容は最新版と異なる場合があります。詳細はお問い合わせください。

目次
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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