Phenylketonuria (PKU) analysis by stage of development, pipeline landscape 2019

Albany, US, 2019-Sep-30 — /EPR Network/ —Market Research Hub (MRH) has actively discoursed a new study titled “Phenylketonuria (PKU)-Pipeline Review, H2 2019”, added to its vast online repository. The main motive of this assessment is to present clear insights about the growth factor experienced across the Phenylketonuria (PKU) during the forecast period. Readers can access vital information associated to prime market facets such as market size, market share, market drivers, future trends, challenges, opportunities and sales channels. Furthermore, the availability of competitive developments including leading market players make the report a rich data source for investors and new entrants.

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Phenylketonuria (PKU) – Pipeline Review, H2 2019


Global Markets Direct’s latest Pharmaceutical and Healthcare disease pipeline guide Phenylketonuria (PKU) – Pipeline Review, H2 2019, provides an overview of the Phenylketonuria (PKU) (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 (PKU) – Pipeline Review, H2 2019, provides comprehensive information on the therapeutics under development for Phenylketonuria (PKU) (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 (PKU) (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, 11 and 1 respectively.

Phenylketonuria (PKU) (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 Directs 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.


– The pipeline guide provides a snapshot of the global therapeutic landscape of Phenylketonuria (PKU) (Metabolic Disorders).
– The pipeline guide reviews pipeline therapeutics for Phenylketonuria (PKU) (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 (PKU) (Metabolic Disorders) therapeutics and enlists all their major and minor projects.
– The pipeline guide evaluates Phenylketonuria (PKU) (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 (PKU) (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 (PKU) (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 its most promising pipeline therapeutics.
– Formulate corrective measures for pipeline projects by understanding Phenylketonuria (PKU) (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.

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Table of Contents
List of Tables
List of Figures
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
Censa Pharmaceuticals Inc
Erytech Pharma SA
Generation Bio Corp
Homology Medicines Inc
MipSalus ApS
Moderna Therapeutics Inc
Nestle Health Science SA
Rubius Therapeutics Inc
SOM Biotech SL
Synlogic Inc
Ultragenyx Pharmaceutical Inc
Phenylketonuria (PKU) – Drug Profiles
APROD-031 – Drug Profile
Product Description
Mechanism Of Action
R&D Progress
BMN-307 – Drug Profile
Product Description
Mechanism Of Action
R&D Progress
CDX-6114 – Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Cell Therapy for Phenylketonuria – Drug Profile
Product Description
Mechanism Of Action
R&D Progress
CNSA-001 – Drug Profile
Product Description
Mechanism Of Action


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