Global Markets Direct’s, ‘Phenylketonuria (PKU) - Pipeline Review, H1 2019’, provides an overview of the Phenylketonuria (PKU) pipeline landscape.
The report provides comprehensive information on the therapeutics under development for Phenylketonuria (PKU), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Phenylketonuria (PKU) and features dormant and discontinued projects.
Global Markets Direct’s report features investigational drugs from across globe covering over 20 therapy areas and nearly 3,000 indications. The report 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. Drug profiles featured in the report undergoes periodic review following a stringent set of processes to ensure that all the profiles are updated with the latest set of information. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.
The report 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.
Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data.
Scope
- The report provides a snapshot of the global therapeutic landscape of Phenylketonuria (PKU)
- The report reviews pipeline therapeutics for Phenylketonuria (PKU) by companies and universities/research institutes based on information derived from company and industry-specific sources
- The report covers pipeline products based on various stages of development ranging from pre-registration till discovery and undisclosed stages
- The report features descriptive drug profiles for the pipeline products which includes, product description, descriptive MoA, R&D brief, licensing and collaboration details & other developmental activities
- The report reviews key players involved Phenylketonuria (PKU) therapeutics and enlists all their major and minor projects
- The report assesses Phenylketonuria (PKU) therapeutics based on drug target, mechanism of action (MoA), route of administration (RoA) and molecule type
- The report summarizes all the dormant and discontinued pipeline projects
- The report reviews latest news related to pipeline therapeutics for Phenylketonuria (PKU)
Reasons to buy
- Gain strategically significant competitor information, analysis, and insights to formulate effective R&D strategies
- Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage
- Identify and understand important and diverse types of therapeutics under development for Phenylketonuria (PKU)
- Identify potential new clients or partners in the target demographic
- Develop strategic initiatives by understanding the focus areas of leading companies
- Plan mergers and acquisitions effectively by identifying key players and it’s most promising pipeline therapeutics
- Devise corrective measures for pipeline projects by understanding Phenylketonuria (PKU) 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
- Modify the therapeutic portfolio by identifying discontinued projects and understanding the factors that drove them from pipeline
Companies mentioned
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 ...
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
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 ...
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
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 ...
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
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 ...
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