Phenylketonuria (PKU) - Pipeline Review, H2 2020

July 2020 | 80 pages | ID: P1DE43C7A04EN
Global Markets Direct

Abstracts

Phenylketonuria (PKU) - Pipeline Review, H2 2020

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.

Contents

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

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

COMPANIES MENTIONED

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