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Phenylketonuria (PKU) - Pipeline Review, H2 2018

Phenylketonuria (PKU) - Pipeline Review, H2 2018

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Phenylketonuria (PKU) - Pipeline Review, H2 2018, 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 2018, 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 Pre-Registration, Phase II, Phase I, Preclinical and Discovery stages are 1, 1, 2, 9 and 3 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 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 (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 it’s 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.


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
BioMarin Pharmaceutical Inc
Censa Pharmaceuticals Inc
Codexis Inc
Erytech Pharma SA
Homology Medicines, Inc.
MipSalus ApS
Rubius Therapeutics Inc
Synlogic Inc
Synthetic Biologics Inc
Ultragenyx Pharmaceutical Inc
Phenylketonuria (PKU) - Drug Profiles
BMN-307 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
CDX-6114 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
CNSA-001 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
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
Gene Therapy for Phenylketonuria - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
HMI-102 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
pegvaliase - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Peptides for Phenylketonuria - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Phelimin - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Recombinant Enzyme for Phenylketonuria - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
RTX-134 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecules for Phenylketonuria - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecules to Activate Phenylalanine Hydroxylase for Phenylketonuria - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
SYN-200 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
SYNB-1618 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Phenylketonuria (PKU) - Dormant Projects
Phenylketonuria (PKU) - Product Development Milestones
Featured News & Press Releases
Jul 09, 2018: Codexis Doses First Subjects in Phase 1a Trial of CDX-
May 24, 2018: BioMarin Receives Standard Approval for Palynziq (pegvaliase-pqpz) Injection for Treatment of Adults with Phenylketonuria (PKU), a Rare Genetic Disease
May 18, 2018: Homology Medicines Presents Data from PKU Gene Therapy Program Demonstrating Long-Term Durability of Treatment in IND-Enabling Studies
Apr 30, 2018: Homology Medicines Announces Upcoming Presentations on its Genetic Medicines Platform at the American Society of Gene & Cell Therapy Meeting
Mar 28, 2018: European Medicines Agency Accepts BioMarin's Marketing Application for Pegvaliase MAA for Treatment of Phenylketonuria
Dec 22, 2017: BioMarin Receives Anticipated Notification of PDUFA Extension for Pegvaliase Biologics License Application (BLA) to May 28,
Sep 14, 2017: FDA Not Currently Planning to Hold Advisory Committee Meeting for BioMarins Pegvaliase Biologics License Application (BLA)
Sep 05, 2017: BioMarin To Present Data On Pegvaliase At 13th International Congress of Inborn Errors of Metabolism
Aug 29, 2017: FDA Accepts BioMarins Pegvaliase Biologics License Application and Grants Priority Review Designation
Jul 31, 2017: MipSalus: Promising Results from Preliminary Safety Study
Jun 30, 2017: BioMarin Submits Pegvaliase Biologics License Application to the U.S. FDA for Treatment of Phenylketonuria
Apr 30, 2017: MipSalus: Multi-dose studies with Phelimin show high efficacy and flexibility
Mar 31, 2016: Promising results with breakthrough treatment of orphan disease PKU
Mar 21, 2016: BioMarin Phase 3 Study of Pegvaliase for Phenylketonuria Meets Primary Endpoint of Blood Phenylalanine Reduction (p0.0001)
Feb 28, 2014: Grant for development of breakthrough treatment of PKU
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 2018
Number of Products under Development by Companies, H2 2018
Products under Development by Companies, H2 2018
Number of Products by Stage and Target, H2 2018
Number of Products by Stage and Mechanism of Action, H2 2018
Number of Products by Stage and Route of Administration, H2 2018
Number of Products by Stage and Molecule Type, H2 2018
Phenylketonuria (PKU) - Pipeline by Agios Pharmaceuticals Inc, H2 2018
Phenylketonuria (PKU) - Pipeline by American Gene Technologies International Inc, H2 2018
Phenylketonuria (PKU) - Pipeline by BioMarin Pharmaceutical Inc, H2 2018
Phenylketonuria (PKU) - Pipeline by Censa Pharmaceuticals Inc, H2 2018
Phenylketonuria (PKU) - Pipeline by Codexis Inc, H2 2018
Phenylketonuria (PKU) - Pipeline by Erytech Pharma SA, H2 2018
Phenylketonuria (PKU) - Pipeline by Homology Medicines, Inc., H2 2018
Phenylketonuria (PKU) - Pipeline by MipSalus ApS, H2 2018
Phenylketonuria (PKU) - Pipeline by Rubius Therapeutics Inc, H2 2018
Phenylketonuria (PKU) - Pipeline by Synlogic Inc, H2 2018
Phenylketonuria (PKU) - Pipeline by Synthetic Biologics Inc, H2 2018
Phenylketonuria (PKU) - Pipeline by Ultragenyx Pharmaceutical Inc, H2 2018
Phenylketonuria (PKU) - Dormant Projects, H2 2018
List of Figures
Number of Products under Development for Phenylketonuria (PKU), H2 2018
Number of Products under Development by Companies, H2 2018
Number of Products by Targets, H2 2018
Number of Products by Stage and Targets, H2 2018
Number of Products by Mechanism of Actions, H2 2018
Number of Products by Stage and Mechanism of Actions, H2 2018
Number of Products by Routes of Administration, H2 2018
Number of Products by Stage and Routes of Administration, H2 2018
Number of Products by Molecule Types, H2 2018
Number of Products by Stage and Molecule Types, H2 2018

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