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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 2020

出版日: | 発行: Global Markets Direct | ページ情報: 英文 80 Pages | 納期: 即納可能 即納可能とは

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フェニルケトン尿症 - パイプライン製品の分析
出版日: 2020年07月31日
発行: Global Markets Direct
ページ情報: 英文 80 Pages
納期: 即納可能 即納可能とは
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  • 概要
  • 図表
  • 目次
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

図表

List of Tables

List of Tables

  • Number of Products under Development for Phenylketonuria (PKU), H2 2020
  • Number of Products under Development by Companies, H2 2020
  • Products under Development by Companies, H2 2020
  • Products under Development by Companies, H2 2020 (Contd..1), H2 2020
  • Number of Products by Stage and Target, H2 2020
  • Number of Products by Stage and Mechanism of Action, H2 2020
  • Number of Products by Stage and Route of Administration, H2 2020
  • Number of Products by Stage and Molecule Type, H2 2020
  • Phenylketonuria (PKU) - Pipeline by Agios Pharmaceuticals Inc, H2 2020
  • Phenylketonuria (PKU) - Pipeline by American Gene Technologies International Inc, H2 2020
  • Phenylketonuria (PKU) - Pipeline by APR Applied Pharma Research SA, H2 2020
  • Phenylketonuria (PKU) - Pipeline by BioMarin Pharmaceutical Inc, H2 2020
  • Phenylketonuria (PKU) - Pipeline by Generation Bio Co, H2 2020
  • Phenylketonuria (PKU) - Pipeline by Homology Medicines Inc, H2 2020
  • Phenylketonuria (PKU) - Pipeline by MipSalus ApS, H2 2020
  • Phenylketonuria (PKU) - Pipeline by Moderna Inc, H2 2020
  • Phenylketonuria (PKU) - Pipeline by Nestle Health Science SA, H2 2020
  • Phenylketonuria (PKU) - Pipeline by Pluvia AS, H2 2020
  • Phenylketonuria (PKU) - Pipeline by PTC Therapeutics Inc, H2 2020
  • Phenylketonuria (PKU) - Pipeline by Sangamo Therapeutics Inc, H2 2020
  • Phenylketonuria (PKU) - Pipeline by SOM Biotech SL, H2 2020
  • Phenylketonuria (PKU) - Pipeline by Synlogic Inc, H2 2020
  • Phenylketonuria (PKU) - Pipeline by Vera Therapeutics Inc, H2 2020
  • Phenylketonuria (PKU) - Dormant Projects, H2 2020
  • Phenylketonuria (PKU) - Discontinued Products, H2 2020

List of Figures

List of Figures

  • Number of Products under Development for Phenylketonuria (PKU), H2 2020
  • Number of Products under Development by Companies, H2 2020
  • Number of Products by Targets, H2 2020
  • Number of Products by Stage and Targets, H2 2020
  • Number of Products by Mechanism of Actions, H2 2020
  • Number of Products by Stage and Mechanism of Actions, H2 2020
  • Number of Products by Routes of Administration, H2 2020
  • Number of Products by Stage and Routes of Administration, H2 2020
  • Number of Products by Molecule Types, H2 2020
  • Number of Products by Stage and Molecule Types, H2 2020
目次
Product Code: GMDHC12414IDB

Summary:

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Phenylketonuria - Pipeline Review, H2 2020, provides an overview of the Phenylketonuria (Metabolic Disorders) pipeline landscape.

Phenylketonuria (also called PKU), is a rare inherited disorder that causes an amino acid called phenylalanine to build up in body. Symptoms include intellectual disability, psychiatric disorders, hyperactivity, skin rashes, poor bone strength and abnormally small head.

Report Highlights:

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Phenylketonuria - Pipeline Review, H2 2020, provides comprehensive information on the therapeutics under development for Phenylketonuria (Metabolic Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.

The Phenylketonuria (Metabolic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Phenylketonuria (PKU) and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase III, Phase II, Phase I, IND/CTA Filed, Preclinical and Discovery stages are 1, 3, 1, 1, 10 and 2 respectively.

Phenylketonuria (Metabolic Disorders) pipeline guide 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. The guide 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. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

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

Scope:

  • The pipeline guide provides a snapshot of the global therapeutic landscape of Phenylketonuria (Metabolic Disorders).
  • The pipeline guide reviews pipeline therapeutics for Phenylketonuria (Metabolic Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources.
  • The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.
  • The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.
  • The pipeline guide reviews key companies involved in Phenylketonuria (Metabolic Disorders) therapeutics and enlists all their major and minor projects.
  • The pipeline guide evaluates Phenylketonuria (Metabolic Disorders) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.
  • The pipeline guide encapsulates all the dormant and discontinued pipeline projects.
  • The pipeline guide reviews latest news related to pipeline therapeutics for Phenylketonuria (Metabolic Disorders)

Reasons to Buy:

  • Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
  • Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.
  • Find and recognize significant and varied types of therapeutics under development for Phenylketonuria (Metabolic Disorders).
  • Classify potential new clients or partners in the target demographic.
  • Develop tactical initiatives by understanding the focus areas of leading companies.
  • Plan mergers and acquisitions meritoriously by identifying key players and it's most promising pipeline therapeutics.
  • Formulate corrective measures for pipeline projects by understanding Phenylketonuria (Metabolic Disorders) 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.
  • Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what 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
  • Phenylketonuria (PKU) - Therapeutics Development
  • Pipeline Overview
  • Pipeline by Companies
  • Products under Development by Companies
  • Phenylketonuria (PKU) - Therapeutics Assessment
  • Assessment by Target
  • Assessment by Mechanism of Action
  • Assessment by Route of Administration
  • Assessment by Molecule Type
  • Phenylketonuria (PKU) - Companies Involved in Therapeutics Development
  • Agios Pharmaceuticals Inc
  • American Gene Technologies International Inc
  • APR Applied Pharma Research SA
  • BioMarin Pharmaceutical Inc
  • Generation Bio Co
  • Homology Medicines Inc
  • MipSalus ApS
  • Moderna Inc
  • Nestle Health Science SA
  • Pluvia AS
  • PTC Therapeutics Inc
  • Sangamo Therapeutics Inc
  • SOM Biotech SL
  • Synlogic Inc
  • Vera Therapeutics Inc
  • Phenylketonuria (PKU) - Drug Profiles
  • AGT-324 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • APROD-031 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Biologic for Phenylketonuria - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Phenylketonuria (PKU) - Dormant Projects
  • Phenylketonuria (PKU) - Discontinued Products
  • Phenylketonuria (PKU) - Product Development Milestones
  • Featured News & Press Releases
  • Appendix
  • Methodology
  • Coverage
  • Secondary Research
  • Primary Research
  • Expert Panel Validation
  • Contact Us
  • Disclaimer
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