+353-1-416-8900REST OF WORLD
+44-20-3973-8888REST OF WORLD
1-917-300-0470EAST COAST U.S
1-800-526-8630U.S. (TOLL FREE)

Phenylketonuria (PKU) - Pipeline Review, H2 2020

  • PDF Icon

    Drug Pipelines

  • 80 Pages
  • July 2020
  • Region: Global
  • Global Markets Direct
  • ID: 5137218
1h Free Analyst Time
1h Free Analyst Time

Speak directly to the analyst to clarify any post sales queries you may have.

Phenylketonuria (PKU) - Pipeline Review, H2 2020

Summary

The publisher'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

The publisher'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 the publisher'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

Introduction
  • 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 ProjectsPhenylketonuria (PKU) - Discontinued Products
Phenylketonuria (PKU) - Product Development Milestones
  • Featured News & Press Releases

Appendix
  • Methodology
  • Coverage
  • Secondary Research
  • Primary Research
  • Expert Panel Validation
  • Contact
  • Disclaimer

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
  • 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

Companies Mentioned (Partial List)

A selection of companies mentioned in this report includes, but is not limited to:

  • 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