Phenylalanine 4 Hydroxylase (Phe 4 Monooxygenase or PAH or EC 1.14.16.1) Development by Therapy Areas and Indications, Stages, MoA, RoA, Molecule Type and Key Players, 2022 Update

March 2022 | 44 pages | ID: P462F5F982A4EN
Global Markets Direct

Abstracts

Phenylalanine 4 Hydroxylase (Phe 4 Monooxygenase or PAH or EC 1.14.16.1) Development by Therapy Areas and Indications, Stages, MoA, RoA, Molecule Type and Key Players, 2022 Update

SUMMARY

Phenylalanine 4 Hydroxylase (Phe 4 Monooxygenase or PAH or EC 1.14.16.1) pipeline Target constitutes close to 12 molecules. Out of which approximately 12 molecules are developed by Companies. The latest report GMDHC22040TDB - Phenylalanine 4 Hydroxylase - Drugs In Development, 2022, outlays comprehensive information on the Phenylalanine 4 Hydroxylase (Phe 4 Monooxygenase or PAH or EC 1.14.16.1) targeted therapeutics, complete with analysis by indications, stage of development, mechanism of action (MoA), route of administration (RoA) and molecule type.

Phenylalanine 4 Hydroxylase (Phe 4 Monooxygenase or PAH or EC 1.14.16.1) - Phenylalanine 4 hydroxylase or Phenylalanine hydroxylase (PAH) is an enzyme that catalyzes the hydroxylation of the aromatic side-chain of phenylalanine to generate tyrosine. The enzyme works with a molecule called tetrahydrobiopterin (BH4) to carry out this chemical reaction. Tyrosine is used to make several types of hormones, certain chemicals that transmit signals in the brain (neurotransmitters), and a pigment called melanin, which gives hair and skin their color. The molecules developed by companies in Phase I, Preclinical and Discovery stages are 2, 5 and 5 respectively. Report covers products from therapy areas Metabolic Disorders which include indications Phenylketonuria (PKU).

Furthermore, this report also reviews key players involved in Phenylalanine 4 Hydroxylase (Phe 4 Monooxygenase or PAH or EC 1.14.16.1) targeted therapeutics development with respective active and dormant or discontinued projects. Driven by data and information sourced from 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.

Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

SCOPE

• The report provides a snapshot of the global therapeutic landscape for Phenylalanine 4 Hydroxylase (Phe 4 Monooxygenase or PAH or EC 1.14.16.1)
• The report reviews Phenylalanine 4 Hydroxylase (Phe 4 Monooxygenase or PAH or EC 1.14.16.1) targeted therapeutics under development 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 in Phenylalanine 4 Hydroxylase (Phe 4 Monooxygenase or PAH or EC 1.14.16.1) targeted therapeutics and enlists all their major and minor projects
• The report assesses Phenylalanine 4 Hydroxylase (Phe 4 Monooxygenase or PAH or EC 1.14.16.1) targeted therapeutics based on 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 and deals related to Phenylalanine 4 Hydroxylase (Phe 4 Monooxygenase or PAH or EC 1.14.16.1) targeted therapeutics

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 the targeted therapy areas and indications for Phenylalanine 4 Hydroxylase (Phe 4 Monooxygenase or PAH or EC 1.14.16.1)
• Identify the use of drugs for target identification and drug repurposing
• 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 Phenylalanine 4 Hydroxylase (Phe 4 Monooxygenase or PAH or EC 1.14.16.1) development landscape
• 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

Contents

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

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

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