Table of Content


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 Intertiol Inc
BioMarin Pharmaceutical Inc
Censa Pharmaceuticals Inc
Codexis Inc
Erytech Pharma SA
Generation Bio Corp
Homology Medicines Inc
MipSalus ApS
Moder 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
mR-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-ebling 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 desigtion 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 tiol 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-Ebling Studies
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer



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


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