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市場調查報告書

溶小體α葡萄糖苷酶:開發中產品分析

Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Pipeline Review, H2 2017

出版商 Global Markets Direct 商品編碼 368724
出版日期 內容資訊 英文 62 Pages
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溶小體α葡萄糖苷酶:開發中產品分析 Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Pipeline Review, H2 2017
出版日期: 2017年10月31日 內容資訊: 英文 62 Pages
簡介

本報告提供以溶小體α葡萄糖苷酶為標的之治療藥開發平台現狀及最新更新的各開發階段比較分析,提供您涵括最新的新聞和發表之企業和研究機關開發中治療藥,治療藥評估,後期階段及中止的計劃等相關資訊。

簡介

  • 調查範圍

溶小體α葡萄糖苷酶 概要

治療藥的開發

  • 開發中的產品:各開發階段
  • 開發中的產品:各治療領域
  • 開發中的產品:不同症狀
  • 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

  • 各作用機制
  • 各給藥途徑
  • 各分子類型

開發治療藥的企業

  • Amicus Therapeutics Inc
  • Audentes Therapeutics Inc
  • Etubics Corp
  • Genzyme Corp
  • greenovation Biotech GmbH
  • Huons Co Ltd
  • JCR Pharmaceuticals Co Ltd
  • Oxyrane Belgium NV
  • Pharming Group NV
  • Sarepta Therapeutics Inc

藥物簡介

暫停中的計劃

開發中止的產品

產品開發的里程碑

  • 主要消息和新聞稿

附錄

圖表

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目錄
Product Code: GMDHC1118TDB

Summary:

Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Lysosomal alpha-glucosidase is an enzyme encoded by the GAA gene. It is essential for the degradation of glygogen to glucose in lysosomes. Defects in this gene lead to glycogen storage disease II or Pompe disease.

Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) pipeline Target constitutes close to 15 molecules. Out of which approximately 12 molecules are developed by companies and remaining by the universities/institutes. The molecules developed by companies in Phase III, Phase II, IND/CTA Filed, Preclinical and Discovery stages are 1, 2, 1, 7 and 1 respectively. Similarly, the universities portfolio in Phase II, Phase I and Preclinical stages comprises 1, 1 and 1 molecules, respectively. Report covers products from therapy areas Metabolic Disorders which include indications Pompe Disease.

The latest report Lysosomal Alpha Glucosidase - Pipeline Review, H2 2017, outlays comprehensive information on the Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) targeted therapeutics, complete with analysis by indications, stage of development, mechanism of action (MoA), route of administration (RoA) and molecule type. It also reviews key players involved in Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) targeted therapeutics development with respective active and dormant or discontinued projects.

The report is built using data and information sourced from 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.

Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

Scope:

  • The report provides a snapshot of the global therapeutic landscape for Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20)
  • The report reviews Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) targeted therapeutics under development 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 in Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) targeted therapeutics and enlists all their major and minor projects
  • The report assesses Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) targeted therapeutics based on 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 and deals related to Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) targeted therapeutics

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 the targeted therapy areas and indications for Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20)
  • Identify the use of drugs for target identification and drug repurposing
  • 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 Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) development landscape
  • 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

Table of Contents

  • Table of Contents
    • List of Tables
    • List of Figures
  • Introduction
    • Global Markets Direct Report Coverage
  • Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Overview
    • Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Therapeutics Development
    • Products under Development by Stage of Development
    • Products under Development by Therapy Area
    • Products under Development by Indication
    • Products under Development by Companies
    • Products under Development by Universities/Institutes
  • Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Therapeutics Assessment
    • Assessment by Mechanism of Action
    • Assessment by Route of Administration
    • Assessment by Molecule Type
  • Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Companies Involved in Therapeutics Development
    • Amicus Therapeutics Inc
    • Audentes Therapeutics Inc
    • Etubics Corp
    • Genzyme Corp
    • greenovation Biotech GmbH
    • JCR Pharmaceuticals Co Ltd
    • NanoMedSyn SAS
    • Oxyrane Belgium NV
    • Pharming Group NV
    • Sarepta Therapeutics Inc
  • Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Drug Profiles
    • Antisense Oligonucleotide to Activate Lysosomal Alpha-Glucosidase for Pompe Disease - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • AT-982 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • ATB-200 + miglustat - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • AVRRD-03 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Gene Therapy 1 to Activate Acid Alpha-Glucosidase for Pompe Disease - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Gene Therapy 2 to Activate Acid Alpha-Glucosidase for Pompe Disease - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Gene Therapy to Activate Acid Alpha-Glucosidase for Pompe Disease - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Gene Therapy to Activate Acid Alpha-Glucosidase for Pompe Disease - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • GZ-402666 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • JR-162 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • MOSS-GAA - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • OXY-2810 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • PGN-004 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Recombinant Alpha Glucosidase Replacement for Pompe Disease - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • VAL-1221 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
  • Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Dormant Products
  • Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Discontinued Products
  • Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Product Development Milestones
    • Featured News & Press Releases
      • Oct 04, 2017: Amicus Therapeutics Announces Additional Positive Data in Pompe Disease Phase 1/2 Study at World Muscle Society
      • Sep 21, 2017: U.S. FDA Grants Orphan Drug Designation for ATB200/AT2221 for Pompe Disease
      • Jul 11, 2017: Valerion Initiates VAL-1221 Dosing in Patients with Pompe Disease
      • May 15, 2017: Amicus Therapeutics Announces Positive Functional Data from Initial Patients in Pompe Phase 1/2 Study
      • Mar 03, 2017: Valerion Therapeutics Demonstrates a New Mechanism for Treating Pompe Disease
      • Feb 23, 2017: JCR to Initiate Development of Jr-162, A New Drug Candidate For Pompe Disease Using J-brain Cargo
      • Feb 15, 2017: Amicus Therapeutics Presents Important New Scientific Findings and Preclinical Data for Pompe Program at WORLDSymposium 2017
      • Dec 08, 2016: Amicus Therapeutics Announces Positive Preliminary Data from Phase 1/2 Study of Novel Treatment Paradigm for Pompe Disease
      • Nov 17, 2016: Pivotal Phase 3 Trial of NeoGAA Investigational Second-Generation Therapy for Pompe Disease to Begin in the UK
      • Nov 04, 2016: Sanofi Genzyme Begins Pivotal Phase 3 Trial of NeoGAA Investigational Second-Generation Therapy for Pompe Disease
      • Mar 03, 2016: Sanofi Genzyme Presents Results from Phase 1/2 Study of Investigational Second-Generation Therapy for Pompe Disease
      • Mar 01, 2016: Amicus Therapeutics Highlights Preclinical Pompe Data at WORLDSymposium 2016
      • Feb 10, 2016: Amicus Therapeutics Announces Oral and Poster Presentations on Chaperone AT2221 at 12th Annual WORLDSymposium 2016
      • Jan 11, 2016: Amicus Therapeutics Provides Update On Novel ERT for Pompe Disease (ATB200 + Chaperone)
      • Dec 22, 2015: Amicus Therapeutics Commences Phase 1/2 Study of Novel ERT for Treatment of Pompe Disease
  • Appendix
    • Methodology
    • Coverage
    • Secondary Research
    • Primary Research
    • Expert Panel Validation
    • Contact Us
  • Disclaimer

List of Tables

  • Number of Products under Development by Stage of Development, H2 2017
  • Number of Products under Development by Therapy Areas, H2 2017
  • Number of Products under Development by Indication, H2 2017
  • Number of Products under Development by Companies, H2 2017
  • Products under Development by Companies, H2 2017
  • Number of Products under Investigation by Universities/Institutes, H2 2017
  • Products under Investigation by Universities/Institutes, H2 2017
  • Number of Products by Stage and Mechanism of Actions, H2 2017
  • Number of Products by Stage and Route of Administration, H2 2017
  • Number of Products by Stage and Molecule Type, H2 2017
  • Pipeline by Amicus Therapeutics Inc, H2 2017
  • Pipeline by Audentes Therapeutics Inc, H2 2017
  • Pipeline by Etubics Corp, H2 2017
  • Pipeline by Genzyme Corp, H2 2017
  • Pipeline by greenovation Biotech GmbH, H2 2017
  • Pipeline by JCR Pharmaceuticals Co Ltd, H2 2017
  • Pipeline by NanoMedSyn SAS, H2 2017
  • Pipeline by Oxyrane Belgium NV, H2 2017
  • Pipeline by Pharming Group NV, H2 2017
  • Pipeline by Sarepta Therapeutics Inc, H2 2017
  • Dormant Projects, H2 2017
  • Discontinued Products, H2 2017

List of Figures

  • Number of Products under Development by Stage of Development, H2 2017
  • Number of Products by Mechanism of Actions, H2 2017
  • Number of Products by Stage and Mechanism of Actions, H2 2017
  • Number of Products by Routes of Administration, H2 2017
  • Number of Products by Stage and Routes of Administration, H2 2017
  • Number of Products by Molecule Types, H2 2017
  • Number of Products by Stage and Molecule Types, H2 2017
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