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フェニルケトン尿症 - パイプライン製品の分析

Phenylketonuria (PKU) - Pipeline Review, H2 2016

発行 Global Markets Direct 商品コード 321900
出版日 ページ情報 英文 51 Pages
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価格
本日の銀行送金レート: 1USD=115.27円で換算しております。

2016年12月31日まで 年末キャンペーン割引: Global Markets Direct社発行レポート
ご注意: ショッピングカートでは適用されませんので、割引価格についてはお問い合わせください

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フェニルケトン尿症 - パイプライン製品の分析 Phenylketonuria (PKU) - Pipeline Review, H2 2016
出版日: 2016年07月13日 ページ情報: 英文 51 Pages
概要

フェニルケトン尿症は希少な遺伝性疾患で、必須アミノ酸であるフェニルアラニンによって引き起こされます。症状は知能障害、精神遅滞、多動、発疹、骨強度の不足、異常に小さな頭部といったものです。

当レポートでは、フェニルケトン尿症に対する治療薬の開発状況について調査分析し、パイプライン製品の概要、治験の段階別の製品の概要、主要企業プロファイル、薬剤のプロファイル、パイプライン製品の最新動向、最新ニュースとプレスリリースなどについて、体系的な情報を提供しています。

目次

イントロダクション

フェニルケトン尿症の概要

治療薬の開発

  • パイプライン製品:概要
  • パイプライン製品:比較分析

開発中の治療薬:企業別

パイプライン製品の概要

  • 後期段階の製品
  • 初期段階の製品

開発中の製品:企業別

治療薬の開発に従事している企業

  • BioMarin Pharmaceutical Inc.
  • Erytech Pharma SA
  • Promethera Biosciences S.A.
  • SOM Innovation Biotech SL

治療薬の評価

  • 単独療法の製品
  • 投与経路別
  • 分子タイプ別

薬剤のプロファイル

  • BMN-165
  • Cell therapy for Phenylketonuria
  • HepaStem
  • sapropterin dihydrochloride
  • SOM-7400

最新のパイプライン情報

開発休止中のプロジェクト

製品開発のマイルストーン

  • 注目のニュースとプレスリリース

付録

図表

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: GMDHC8233IDB

Summary

Global Markets Direct's, 'Phenylketonuria (PKU) - Pipeline Review, H2 2016', provides an overview of the Phenylketonuria (PKU) pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Phenylketonuria (PKU), 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 Phenylketonuria (PKU) 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 Phenylketonuria (PKU)
  • The report reviews pipeline therapeutics for Phenylketonuria (PKU) 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 Phenylketonuria (PKU) therapeutics and enlists all their major and minor projects
  • The report assesses Phenylketonuria (PKU) 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 Phenylketonuria (PKU)

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 Phenylketonuria (PKU)
  • 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 Phenylketonuria (PKU) 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
  • Phenylketonuria (PKU) Overview
  • Therapeutics Development
    • Pipeline Products for Phenylketonuria (PKU) - Overview
  • Phenylketonuria (PKU) - Therapeutics under Development by Companies
  • Phenylketonuria (PKU) - Pipeline Products Glance
    • Late Stage Products
    • Early Stage Products
  • Phenylketonuria (PKU) - Products under Development by Companies
  • Phenylketonuria (PKU) - Companies Involved in Therapeutics Development
    • American Gene Technologies International Inc.
    • BioMarin Pharmaceutical Inc.
    • Codexis, Inc.
    • Dimension Therapeutics, Inc.
    • Erytech Pharma SA
    • SOM Innovation Biotech SL
    • Synthetic Biologics, Inc.
  • Phenylketonuria (PKU) - 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
    • DTX-501 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Gene Therapy for Phenylketonuria - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • pegvaliase - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Proteins to Activate Phenylalanine Hydroxylase for Phenylketonuria - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Recombinant Enzyme for Phenylketonuria - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Recombinant Phenylalanine-4-Hydroxylase Replacement for Phenylketonuria - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Recombinant Phenylalanine-4-Hydroxylase Replacement for Phenylketonuria - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • SOM-7400 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • SYN-200 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • SYNB-2010 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
  • Phenylketonuria (PKU) - Dormant Projects
  • Phenylketonuria (PKU) - Product Development Milestones
    • Featured News & Press Releases
      • Mar 21, 2016: BioMarin Phase 3 Study of Pegvaliase for Phenylketonuria Meets Primary Endpoint of Blood Phenylalanine Reduction (p0.0001)
      • Jun 05, 2013: BioMarin Initiates Phase III Trial For PEG-PAL For Treatment Of Phenylketonuria
      • Sep 26, 2012: BioMarin Pharma To Initiate Phase III Program For PEG-PAL In Second Quarter Of 2013
      • Aug 02, 2010: BioMarin Reports Encouraging Preliminary Safety And Efficacy Trends In Phase II Clinical Study Of PEG-PAL In Phenylketonuria
      • Sep 22, 2009: BioMarin Initiates Phase II Clinical Study Of PEG-PAL In PKU
      • Jun 09, 2009: Results From Phase 1 Clinical Study Of PEG-PAL In PKU And Update On Phase 2 Clinical Study
      • May 20, 2008: BioMarin Initiates Phase 1 Clinical Study Of PEG-PAL In PKU
      • Nov 27, 2007: BioMarin Files Investigational New Drug Application For PEG-PAL For The Treatment Of PKU
      • May 16, 2005: BioMarin And Serono Form Strategic Alliance For The Development And Commercialization Of Phenoptin And Phenylase
  • Appendix
    • Methodology
    • Coverage
    • Secondary Research
    • Primary Research
    • Expert Panel Validation
    • Contact Us
    • Disclaimer

List of Tables

  • Number of Products under Development for Phenylketonuria (PKU), H2 2016
  • Number of Products under Development by Companies, H2 2016
  • Comparative Analysis by Late Stage Development, H2 2016
  • Comparative Analysis by Early Stage Development, H2 2016
  • Products under Development by Companies, H2 2016
  • Phenylketonuria (PKU) - Pipeline by American Gene Technologies International Inc., H2 2016
  • Phenylketonuria (PKU) - Pipeline by BioMarin Pharmaceutical Inc., H2 2016
  • Phenylketonuria (PKU) - Pipeline by Codexis, Inc., H2 2016
  • Phenylketonuria (PKU) - Pipeline by Dimension Therapeutics, Inc., H2 2016
  • Phenylketonuria (PKU) - Pipeline by Erytech Pharma SA, H2 2016
  • Phenylketonuria (PKU) - Pipeline by SOM Innovation Biotech SL, H2 2016
  • Phenylketonuria (PKU) - Pipeline by Synthetic Biologics, 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
  • Phenylketonuria (PKU) - Dormant Projects, H2 2016

List of Figures

  • Number of Products under Development for Phenylketonuria (PKU), H2 2016
  • Number of Products under Development by Companies, 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 Mechanism of Actions, H2 2016
  • Number of Products by Stage and 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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