Phenylalanine 4 Hydroxylase (Phe 4 Monooxygenase or PAH or EC 1.14.16.1) - Drugs in Development, 2021

March 2021 | 65 pages | ID: PDA403AA5EC2EN
Global Markets Direct

Abstracts

Phenylalanine 4 Hydroxylase (Phe 4 Monooxygenase or PAH or EC 1.14.16.1) - Drugs in Development, 2021

SUMMARY

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.

Phenylalanine 4 Hydroxylase (Phe 4 Monooxygenase or PAH or EC 1.14.16.1) pipeline Target constitutes close to 10 molecules. The molecules developed by companies in Phase II, Phase I, Preclinical and Discovery stages are 2, 1, 6 and 1 respectively. Report covers products from therapy areas Metabolic Disorders which include indications Phenylketonuria (PKU) and Phenylalanine Hydroxylase Deficiencies.

The latest report Phenylalanine 4 Hydroxylase - Drugs in Development, 2021, 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. It 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.

The report is built using 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
BioMarin Pharmaceutical Inc
Homology Medicines Inc
Moderna Inc
Nestle Health Science SA
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
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
mRNA-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 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
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 Designation for PKU investigational 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 investigational 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 investigational pku gene editing program, demonstrating phenotypic correction and molecular confirmation of editing precision
Oct 21, 2019: Homology Medicines presents data from investigational 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 Tables

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

List Of Figures

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