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Market Research Report

Phenylketonuria (PKU) - Pipeline Review, H1 2019

Published by Global Markets Direct Product code 321900
Published Content info 67 Pages
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Phenylketonuria (PKU) - Pipeline Review, H1 2019
Published: January 31, 2019 Content info: 67 Pages
Description

Summary:

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Phenylketonuria (PKU) - Pipeline Review, H1 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, H1 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 Pre-Registration, Phase II, Phase I, Preclinical and Discovery stages are 1, 2, 1, 12 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 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.
Table of Contents
Product Code: GMDHC11100IDB

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
    • BioMarin Pharmaceutical Inc
    • Censa Pharmaceuticals Inc
    • Codexis Inc
    • Erytech Pharma SA
    • Generation Bio Corp
    • Homology Medicines Inc
    • MipSalus ApS
    • Moderna Therapeutics Inc
    • 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
    • Cell Therapy for Phenylketonuria - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • CNSA-001 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Gene Therapy for Phenylketonuria and Progressive Familial Intrahepatic Cholestasis - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Gene Therapy to Activate Phenylalanine Hydroxylase for Phenylketonuria - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • HMI-102 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • HMI-103 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • mRNA-3283 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • pegvaliase - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Phelimin - 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
    • UX-501 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
  • Phenylketonuria (PKU) - Dormant Projects
  • Phenylketonuria (PKU) - Product Development Milestones
    • Featured News & Press Releases
      • Jan 07, 2019: Retrophin provides update on phenylketonuria drug CNSA-001
      • Jan 07, 2019: BioMarin highlights Palynziq at 37th Annual J.P. Morgan Healthcare Conference in San Francisco
      • Jan 07, 2019: Rubius Therapeutics provides update on its phenylketonuria drug candidate RTX-134 at the 37th Annual J.P. Morgan Healthcare Conference
      • Jan 07, 2019: Homology Medicines achieves 2018 goals and prepares PKU Gene Therapy Program for the clinic with initial data expected in 2019
      • Jan 07, 2019: Homology Medicines advances first gene editing development candidate into IND-enabling studies for pediatric patients with phenylketonuria
      • Nov 08, 2018: Codexis announces results of phase 1a clinical trial with CDX-6114
      • Nov 07, 2018: BioMarin provides update on Palynziq at at R&D Day in New York
      • Nov 07, 2018: BioMarin provides update on BMN 307, a pre-clinical gene therapy for PKU
      • Oct 18, 2018: American Gene Technologies granted FDA orphan drug designation for phenylketonuria
      • Oct 18, 2018: Homology Medicines Presents Long-Term Efficacy Data of a Single Dose of Gene Therapy Development Candidate for Treatment of Phenylketonuria
      • Jul 11, 2018: Codexis Appoints Dr. Hicham Alaoui, Veteran Drug Discoverer, as Vice President, Biotherapeutics Research & Development
      • Jul 09, 2018: Codexis Doses First Subjects in Phase 1a Trial of CDX-6114
      • Jul 05, 2018: Homology Medicines to present at the National PKU Alliance Conference
      • 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
  • Appendix
    • Methodology
    • Coverage
    • Secondary Research
    • Primary Research
    • Expert Panel Validation
    • Contact Us
  • Disclaimer

List of Tables

  • Number of Products under Development for Phenylketonuria (PKU), H1 2019
  • Number of Products under Development by Companies, H1 2019
  • Products under Development by Companies, H1 2019
  • Number of Products by Stage and Target, H1 2019
  • Number of Products by Stage and Mechanism of Action, H1 2019
  • Number of Products by Stage and Route of Administration, H1 2019
  • Number of Products by Stage and Molecule Type, H1 2019
  • Phenylketonuria (PKU) - Pipeline by Agios Pharmaceuticals Inc, H1 2019
  • Phenylketonuria (PKU) - Pipeline by American Gene Technologies International Inc, H1 2019
  • Phenylketonuria (PKU) - Pipeline by BioMarin Pharmaceutical Inc, H1 2019
  • Phenylketonuria (PKU) - Pipeline by Censa Pharmaceuticals Inc, H1 2019
  • Phenylketonuria (PKU) - Pipeline by Codexis Inc, H1 2019
  • Phenylketonuria (PKU) - Pipeline by Erytech Pharma SA, H1 2019
  • Phenylketonuria (PKU) - Pipeline by Generation Bio Corp, H1 2019
  • Phenylketonuria (PKU) - Pipeline by Homology Medicines Inc, H1 2019
  • Phenylketonuria (PKU) - Pipeline by MipSalus ApS, H1 2019
  • Phenylketonuria (PKU) - Pipeline by Moderna Therapeutics Inc, H1 2019
  • Phenylketonuria (PKU) - Pipeline by Rubius Therapeutics Inc, H1 2019
  • Phenylketonuria (PKU) - Pipeline by Synlogic Inc, H1 2019
  • Phenylketonuria (PKU) - Pipeline by Synthetic Biologics Inc, H1 2019
  • Phenylketonuria (PKU) - Pipeline by Ultragenyx Pharmaceutical Inc, H1 2019
  • Phenylketonuria (PKU) - Dormant Projects, H1 2019

List of Figures

  • Number of Products under Development for Phenylketonuria (PKU), H1 2019
  • Number of Products under Development by Companies, H1 2019
  • Number of Products by Targets, H1 2019
  • Number of Products by Stage and Targets, H1 2019
  • Number of Products by Mechanism of Actions, H1 2019
  • Number of Products by Stage and Mechanism of Actions, H1 2019
  • Number of Products by Routes of Administration, H1 2019
  • Number of Products by Stage and Routes of Administration, H1 2019
  • Number of Products by Molecule Types, H1 2019
  • Number of Products by Stage and Molecule Types, H1 2019
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