List of Tables
List of Figures
Introduction
Global Markets Direct Report Coverage
Phenylalanine 4 Hydroxylase (Phe 4 Monooxygenase or PAH or EC 1.14.16.1) - Overview
Phenylalanine 4 Hydroxylase (Phe 4 Monooxygenase or PAH or EC 1.14.16.1) - Therapeutics Development
Products under Development by Stage of Development
Products under Development by Therapy Area
Products under Development by Indication
Products under Development by Companies
Phenylalanine 4 Hydroxylase (Phe 4 Monooxygenase or PAH or EC 1.14.16.1) - Therapeutics Assessment
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Phenylalanine 4 Hydroxylase (Phe 4 Monooxygenase or PAH or EC 1.14.16.1) - Companies Involved in Therapeutics Development
Agios Pharmaceuticals Inc
American Gene Technologies International Inc
Castle Creek Biosciences Inc
CommBio Therapeutics Co Ltd
Evox Therapeutics Ltd
Homology Medicines Inc
iECURE
Moderna Inc
Nestle Health Science
Pluvia AS
Ultragenyx Pharmaceutical Inc
Vera Therapeutics Inc
Phenylalanine 4 Hydroxylase (Phe 4 Monooxygenase or PAH or EC 1.14.16.1) - Drug Profiles
AGT-324 - Drug Profile
Product Description
Mechanism Of Action
History of Events
CDX-6114 - Drug Profile
Product Description
Mechanism Of Action
History of Events
Gene Therapy for Phenylketonuria - Drug Profile
Product Description
Mechanism Of Action
Gene Therapy to Target Phenylalanine Hydroxylase for Phenylketonuria - Drug Profile
Product Description
Mechanism Of Action
History of Events
Gene-Modified Cell Therapy to Activate PAH for Phenylketonuria - Drug Profile
Product Description
Mechanism Of Action
HMI-103 - Drug Profile
Product Description
Mechanism Of Action
History of Events
mRNA-3283 - Drug Profile
Product Description
Mechanism Of Action
History of Events
Oligonucleotides to Activate PAH for Phenylketonuria - Drug Profile
Product Description
Mechanism Of Action
History of Events
PAH - Drug Profile
Product Description
Mechanism Of Action
Phenylketonuria - Drug Profile
Product Description
Mechanism Of Action
Small Molecules to Activate Phenylalanine Hydroxylase for Phenylketonuria - Drug Profile
Product Description
Mechanism Of Action
UX-501 - Drug Profile
Product Description
Mechanism Of Action
History of Events
Phenylalanine 4 Hydroxylase (Phe 4 Monooxygenase or PAH or EC 1.14.16.1) - Dormant Products
Phenylalanine 4 Hydroxylase (Phe 4 Monooxygenase or PAH or EC 1.14.16.1) - Discontinued Products
Phenylalanine 4 Hydroxylase (Phe 4 Monooxygenase or PAH or EC 1.14.16.1) - Product Development Milestones
Featured News & Press Releases
Oct 12, 2021: Homology Medicines announces world's first gene editing clinical trial for PKU
Mar 10, 2020: American Gene Technologies to present at 2020 Inborn Errors of Metabolism Drug Development Summit in Boston
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
Oct 18, 2018: American Gene Technologies granted FDA orphan drug designation for 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
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer

List of Tables
Number of Products under Development by Stage of Development, 2022
Number of Products under Development by Therapy Areas, 2022
Number of Products under Development by Indication, 2022
Number of Products under Development by Companies, 2022
Products under Development by Companies, 2022
Number of Products by Stage and Mechanism of Actions, 2022
Number of Products by Stage and Route of Administration, 2022
Number of Products by Stage and Molecule Type, 2022
Pipeline by Agios Pharmaceuticals Inc, 2022
Pipeline by American Gene Technologies International Inc, 2022
Pipeline by Castle Creek Biosciences Inc, 2022
Pipeline by CommBio Therapeutics Co Ltd, 2022
Pipeline by Evox Therapeutics Ltd, 2022
Pipeline by Homology Medicines Inc, 2022
Pipeline by iECURE, 2022
Pipeline by Moderna Inc, 2022
Pipeline by Nestle Health Science, 2022
Pipeline by Pluvia AS, 2022
Pipeline by Ultragenyx Pharmaceutical Inc, 2022
Pipeline by Vera Therapeutics Inc, 2022
Dormant Projects, 2022
Discontinued Products, 2022

List of Figures
Number of Products under Development by Stage of Development, 2022
Number of Products by Mechanism of Actions, 2022
Number of Products by Stage and Mechanism of Actions, 2022
Number of Products by Routes of Administration, 2022
Number of Products by Stage and Routes of Administration, 2022
Number of Products by Molecule Types, 2022
Number of Products by Stage and Molecule Types, 2022

Companies Mentioned
• Agios Pharmaceuticals Inc
• American Gene Technologies International Inc
• Castle Creek Biosciences Inc
• CommBio Therapeutics Co Ltd
• Evox Therapeutics Ltd
• Homology Medicines Inc
• iECURE
• Moderna Inc
• Nestle Health Science
• Pluvia AS
• Ultragenyx Pharmaceutical Inc
• Vera Therapeutics Inc