Summary
Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Phenylketonuria (PKU) - Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update, provides an overview of the Phenylketonuria (PKU) (Metabolic Disorders) pipeline landscape.
Phenylketonuria (also called PKU) is a rare inherited disorder that causes an amino acid called phenylalanine to build up in body. Symptoms include intellectual disability, psychiatric disorders, hyperactivity, skin rashes, poor bone strength and abnormally small head.
Report Highlights
Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Phenylketonuria (PKU) - Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update, provides comprehensive information on the therapeutics under development for Phenylketonuria (PKU) (Metabolic Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.
The Phenylketonuria (PKU) (Metabolic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Phenylketonuria (PKU) and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase III, Phase II, Phase I, Preclinical and Discovery stages are 2, 1, 3, 11 and 8 respectively.
Phenylketonuria (PKU) (Metabolic Disorders) pipeline guide helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage. The guide is built using data and information sourced from Global Markets Direct’s 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. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.
Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.
Scope
- The pipeline guide provides a snapshot of the global therapeutic landscape of Phenylketonuria (PKU) (Metabolic Disorders).
- The pipeline guide reviews pipeline therapeutics for Phenylketonuria (PKU) (Metabolic Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources.
- The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.
- The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.
- The pipeline guide reviews key companies involved in Phenylketonuria (PKU) (Metabolic Disorders) therapeutics and enlists all their major and minor projects.
- The pipeline guide evaluates Phenylketonuria (PKU) (Metabolic Disorders) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.
- The pipeline guide encapsulates all the dormant and discontinued pipeline projects.
- The pipeline guide reviews latest news related to pipeline therapeutics for Phenylketonuria (PKU) (Metabolic Disorders)
Reasons to Buy
- Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
- Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.
- Find and recognize significant and varied types of therapeutics under development for Phenylketonuria (PKU) (Metabolic Disorders).
- Classify potential new clients or partners in the target demographic.
- Develop tactical initiatives by understanding the focus areas of leading companies.
- Plan mergers and acquisitions meritoriously by identifying key players and it’s most promising pipeline therapeutics.
- Formulate corrective measures for pipeline projects by understanding Phenylketonuria (PKU) (Metabolic Disorders) pipeline depth and focus of Indication therapeutics.
- 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.
- Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline.
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Table of Contents
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 International Inc
BioMarin Pharmaceutical Inc
Castle Creek Biosciences Inc
CommBio Therapeutics Co Ltd
Evox Therapeutics Ltd
Generation Bio Co
Hepatx Corp
Homology Medicines Inc
iECURE
Jnana Therapeutics Inc
Maze Therapeutics Inc
MipSalus ApS
Moderna Inc
Nestle Health Science
Peg-Bio BioPharma Co Ltd
Pluvia AS
Prosit Sole Biotechnology (Beijing) Co Ltd
PTC Therapeutics Inc
Relief Therapeutics Holding AG
SOM Biotech SL
Synlogic Inc
Ultragenyx Pharmaceutical Inc
Vera Therapeutics Inc
Phenylketonuria (PKU) - Drug Profiles
AGT-324 - Drug Profile
Product Description
Mechanism Of Action
History of Events
APROD-031 - 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
HX-003 - Drug Profile
Product Description
Mechanism Of Action
JNT-517 - Drug Profile
Product Description
Mechanism Of Action
mRNA-3283 - Drug Profile
Product Description
Mechanism Of Action
Oligonucleotides to Activate PAH for Phenylketonuria - Drug Profile
Product Description
Mechanism Of Action
History of Events
PAH - Drug Profile
Product Description
Mechanism Of Action
pegvaliase - Drug Profile
Product Description
Mechanism Of Action
History of Events
Phelimin - Drug Profile
Product Description
Mechanism Of Action
History of Events
Phenylketonuria - Drug Profile
Product Description
Mechanism Of Action
PJ-008 - Drug Profile
Product Description
Mechanism Of Action
PKU - Drug Profile
Product Description
Mechanism Of Action
PSP-007 - Drug Profile
Product Description
Mechanism Of Action
sepiapterin - Drug Profile
Product Description
Mechanism Of Action
History of Events
Small Molecule for Phenylketonuria - Drug Profile
Product Description
Mechanism Of Action
Small Molecules to Activate Phenylalanine Hydroxylase for Phenylketonuria - Drug Profile
Product Description
Mechanism Of Action
SOM-1311 - Drug Profile
Product Description
Mechanism Of Action
SYNB-1618 - Drug Profile
Product Description
Mechanism Of Action
History of Events
SYNB-1934 - Drug Profile
Product Description
Mechanism Of Action
History of Events
UX-501 - Drug Profile
Product Description
Mechanism Of Action
History of Events
Phenylketonuria (PKU) - Dormant Projects
Phenylketonuria (PKU) - Discontinued Products
Phenylketonuria (PKU) - Product Development Milestones
Featured News & Press Releases
Feb 23, 2022: Jnana Therapeutics to advance JNT-517 as development candidate and potential first-in-class oral treatment for PKU
Nov 22, 2021: Synlogic presents data demonstrating reductions in plasma phenylalanine levels in patients with phenylketonuria treated with SYNB1934
Nov 22, 2021: Synlogic presents data demonstrating reductions in plasma phenylalanine levels in patients with phenylketonuria treated with SYNB1618
Nov 10, 2021: Synlogic announces presentation of data on SYNB1934 at 14th International Congress of Inborn Errors of Metabolism Meeting
Nov 10, 2021: Synlogic announces presentation of data on SYNB1618 at 14th International Congress of Inborn Errors of Metabolism Meeting
Oct 28, 2021: Synlogic announces Nature Communications publication highlighting use of synthetic biotic platform to optimize therapies for phenylketonuria
Oct 12, 2021: Homology Medicines announces world’s first gene editing clinical trial for PKU
Sep 11, 2021: Synlogic presents at Global PKU Patient Meeting
Jul 22, 2021: Synlogic publishes papers in Nature journals demonstrating proof-of-mechanism and potential of synthetic biotic platform for the treatment of phenylketonuria (PKU)
Jul 15, 2021: Synlogic provides update on SYNB1618 , a next-generation strain for the treatment of Phenylketonuria (PKU)
Jul 15, 2021: Synlogic announces initiation of phase 1 study of SYNB1934, a next-generation strain for the treatment of Phenylketonuria (PKU)
Jun 15, 2021: Synlogic presents additional preclinical data on therapeutic candidate SYNB1934 for Phenylketonuria (PKU) at Synthetic Biology: Engineering, Evolution & Design (SEED) Conference
Apr 13, 2021: Synlogic presents data demonstrating activity of a solid oral formulation of SYNB1618 at American College of Medical Genetics (ACMG) annual meeting
Oct 07, 2020: BioMarin, pioneer in rare disease treatments for Phenylketonuria (PKU), receives FDA approval of label expansion to allow maximum dose of 60 mg for Palynziq (pegvaliase-pqpz) injection for treatment of adults with PKU
Mar 26, 2020: FDA grants Orphan Drug Designation to APR-OD031 for the treatment of phenylketonuria
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer
List of Tables
Number of Products under Development for Phenylketonuria (PKU), 2022
Number of Products under Development by Companies, 2022
Products under Development by Companies, 2022
Products under Development by Companies, 2022 (Contd..1)
Number of Products by Stage and Target, 2022
Number of Products by Stage and Mechanism of Action, 2022
Number of Products by Stage and Route of Administration, 2022
Number of Products by Stage and Molecule Type, 2022
Phenylketonuria (PKU) - Pipeline by Agios Pharmaceuticals Inc, 2022
Phenylketonuria (PKU) - Pipeline by American Gene Technologies International Inc, 2022
Phenylketonuria (PKU) - Pipeline by BioMarin Pharmaceutical Inc, 2022
Phenylketonuria (PKU) - Pipeline by Castle Creek Biosciences Inc, 2022
Phenylketonuria (PKU) - Pipeline by CommBio Therapeutics Co Ltd, 2022
Phenylketonuria (PKU) - Pipeline by Evox Therapeutics Ltd, 2022
Phenylketonuria (PKU) - Pipeline by Generation Bio Co, 2022
Phenylketonuria (PKU) - Pipeline by Hepatx Corp, 2022
Phenylketonuria (PKU) - Pipeline by Homology Medicines Inc, 2022
Phenylketonuria (PKU) - Pipeline by iECURE, 2022
Phenylketonuria (PKU) - Pipeline by Jnana Therapeutics Inc, 2022
Phenylketonuria (PKU) - Pipeline by Maze Therapeutics Inc, 2022
Phenylketonuria (PKU) - Pipeline by MipSalus ApS, 2022
Phenylketonuria (PKU) - Pipeline by Moderna Inc, 2022
Phenylketonuria (PKU) - Pipeline by Nestle Health Science, 2022
Phenylketonuria (PKU) - Pipeline by Peg-Bio BioPharma Co Ltd, 2022
Phenylketonuria (PKU) - Pipeline by Pluvia AS, 2022
Phenylketonuria (PKU) - Pipeline by Prosit Sole Biotechnology (Beijing) Co Ltd, 2022
Phenylketonuria (PKU) - Pipeline by PTC Therapeutics Inc, 2022
Phenylketonuria (PKU) - Pipeline by Relief Therapeutics Holding AG, 2022
Phenylketonuria (PKU) - Pipeline by SOM Biotech SL, 2022
Phenylketonuria (PKU) - Pipeline by Synlogic Inc, 2022
Phenylketonuria (PKU) - Pipeline by Ultragenyx Pharmaceutical Inc, 2022
Phenylketonuria (PKU) - Pipeline by Vera Therapeutics Inc, 2022
Phenylketonuria (PKU) - Dormant Projects, 2022
Phenylketonuria (PKU) - Discontinued Products, 2022
List of Figures
Number of Products under Development for Phenylketonuria (PKU), 2022
Number of Products under Development by Companies, 2022
Number of Products by Targets, 2022
Number of Products by Stage and Targets, 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 Top 10 Molecule Types, 2022
Number of Products by Stage and Top 10 Molecule Types, 2022
