Table of Content


List of Tables
List of Figures
Introduction
Global Markets Direct Report Coverage
Phenylalanine 4 Hydroxylase (Phe 4 Monooxygese or PAH or EC 1.14.16.1) - Overview
Phenylalanine 4 Hydroxylase (Phe 4 Monooxygese 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 Monooxygese 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 Monooxygese or PAH or EC 1.14.16.1) - Companies Involved in Therapeutics Development
Agios Pharmaceuticals Inc
American Gene Technologies Intertiol Inc
BioMarin Pharmaceutical Inc
Homology Medicines Inc
Moder Inc
Nestle Health Science SA
Pluvia AS
Ultragenyx Pharmaceutical Inc
Vera Therapeutics Inc
Phenylalanine 4 Hydroxylase (Phe 4 Monooxygese or PAH or EC 1.14.16.1) - Drug Profiles
AGT-324 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
BMN-307 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
CDX-6114 - 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
Oligonucleotides to Activate PAH 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
Small Molecules to Target PAH for Phenylketonuria - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
UX-501 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Phenylalanine 4 Hydroxylase (Phe 4 Monooxygese or PAH or EC 1.14.16.1) - Dormant Products
Phenylalanine 4 Hydroxylase (Phe 4 Monooxygese or PAH or EC 1.14.16.1) - Discontinued Products
Phenylalanine 4 Hydroxylase (Phe 4 Monooxygese or PAH or EC 1.14.16.1) - Product Development Milestones
Featured News & Press Releases
Nov 06, 2020: Homology Medicines announces presentation of positive data from the dose-escalation phase of the pheNIX Gene Therapy trial for adults with PKU
Nov 05, 2020: Homology Medicines announces upcoming oral presentation on pheNIX gene therapy clinical trial for adults with PKU
Oct 02, 2020: BioMarin, pioneer in phenylketonuria (PKU) and gene therapy, receives FDA Fast Track Desigtion for PKU investigatiol gene therapy, BMN 307
Sep 24, 2020: BioMarin, pioneer in phenylketonuria (PKU) and gene therapy, doses first participant in global PHEARLESS phase 1/2 study of BMN 307 gene therapy
May 26, 2020: Homology Medicines announces Peer-Reviewed Publication describing molecular characterization of precise In Vivo Nuclease-Free gene editing with PKU program
May 04, 2020: Homology Medicines announces Publication in Genetic Engineering & Biotechnology News on dual gene therapy and nuclease-free gene editing platform for PKU
Apr 29, 2020: Homology Medicines announces presentations at upcoming American Society for Gene & Cell Therapy Annual Meeting
Mar 16, 2020: Homology Medicines announces peer-reviewed publication of HMI-102 investigatiol gene therapy demonstrating restoration of normal metabolic pathway in PKU disease model
Mar 10, 2020: American Gene Technologies to present at 2020 Inborn Errors of Metabolism Drug Development Summit in Boston
Jan 13, 2020: BioMarin, pioneer in phenylketonuria, to begin clinical trial with BMN 307 gene therapy
Dec 17, 2019: Homology Medicines announces encouraging initial clinical data from its pheNIX Gene Therapy Trial for PKU
Dec 16, 2019: Homology Medicines announces peer-reviewed publication of five-year retrospective study conducted in collaboration with two PKU Key Opinion Leaders demonstrating substantially elevated Phe levels in adults with classical PKU on standard of care
Dec 03, 2019: Homology Medicines announces appointment of Gabriel M. Cohn, M.D., as chief medical officer
Oct 25, 2019: Homology Medicines presents preclinical data from its investigatiol pku gene editing program, demonstrating phenotypic correction and molecular confirmation of editing precision
Oct 21, 2019: Homology Medicines presents data from investigatiol MLD gene therapy program HMI-102 at the American Society of Human Genetics (ASHG) 2019 Meeting
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer



List of Figures


Number of Products under Development by Stage of Development, 2021
Number of Products under Development by Top 10 Indications, 2021
Number of Products by Mechanism of Actions, 2021
Number of Products by Stage and Mechanism of Actions, 2021
Number of Products by Routes of Administration, 2021
Number of Products by Stage and Routes of Administration, 2021
Number of Products by Molecule Types, 2021
Number of Products by Stage and Molecule Types, 2021


List of Tables


Number of Products under Development by Stage of Development, 2021
Number of Products under Development by Therapy Areas, 2021
Number of Products under Development by Indication, 2021
Number of Products under Development by Companies, 2021
Products under Development by Companies, 2021
Number of Products by Stage and Mechanism of Actions, 2021
Number of Products by Stage and Route of Administration, 2021
Number of Products by Stage and Molecule Type, 2021
Pipeline by Agios Pharmaceuticals Inc, 2021
Pipeline by American Gene Technologies International Inc, 2021
Pipeline by BioMarin Pharmaceutical Inc, 2021
Pipeline by Homology Medicines Inc, 2021
Pipeline by Moderna Inc, 2021
Pipeline by Nestle Health Science SA, 2021
Pipeline by Pluvia AS, 2021
Pipeline by Ultragenyx Pharmaceutical Inc, 2021
Pipeline by Vera Therapeutics Inc, 2021
Dormant Projects, 2021
Discontinued Products, 2021