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Phenylketonuria (PKU) (Metabolic Disorder) - Drugs in Development, 2021

March 2021 | 91 pages | ID: P5FB6BCC7604EN
Global Markets Direct

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Phenylketonuria (PKU) (Metabolic Disorder) - Drugs in Development, 2021

SUMMARY

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Phenylketonuria - Drugs in Development, 2021, provides an overview of the Phenylketonuria (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 - Drugs in Development, 2021, provides comprehensive information on the therapeutics under development for Phenylketonuria (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 (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 1, 4, 2, 11 and 4 respectively.

Phenylketonuria (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 (Metabolic Disorders).
  • The pipeline guide reviews pipeline therapeutics for Phenylketonuria (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 (Metabolic Disorders) therapeutics and enlists all their major and minor projects.
  • The pipeline guide evaluates Phenylketonuria (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 (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 (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 (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.
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
APR Applied Pharma Research SA
BioMarin Pharmaceutical Inc
Evox Therapeutics Ltd
Generation Bio Co
Homology Medicines Inc
MipSalus ApS
Moderna Inc
Nestle Health Science SA
Peg-Bio BioPharma Co Ltd
Pluvia AS
Prosit Sole Biotechnology (Beijing) Co Ltd
PTC Therapeutics Inc
Sangamo Therapeutics Inc
SOM Biotech SL
Synlogic Inc
Ultragenyx Pharmaceutical Inc
Vera Therapeutics Inc
Phenylketonuria (PKU) - Drug Profiles
AGT-324 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
APROD-031 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Biologic for Phenylketonuria - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Biologics for Phenylketonuria - 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
Gene Therapy for Phenylketonuria and Progressive Familial Intrahepatic Cholestasis - 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
pegvaliase - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Phelimin - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
PJ-008 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
PSP-007 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
PTC-923 - 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
SOM-1311 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
ST-101 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
SYNB-1618 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
UX-501 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Phenylketonuria (PKU) - Dormant Projects
Phenylketonuria (PKU) - Discontinued Products
Phenylketonuria (PKU) - Product Development Milestones
Featured News & Press Releases
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
Mar 10, 2020: American Gene Technologies to present at 2020 Inborn Errors of Metabolism Drug Development Summit in Boston
Dec 11, 2019: Synlogic bridging study data with solid oral formulation of SYNB1618, a synthetic biotic approach to treat Phenylketonuria, demonstrate improved tolerability over early liquid formulation and guide to next stage of clinical development
Dec 03, 2019: Censa Pharmaceuticals announces CNSA- 001 met primary and secondary endpoints in phase 2 trial in patients with PKU
Sep 04, 2019: Synlogic presents data from phase 1/2a study of SYNB1618 at the Annual Symposium of the Society for the Study of Inborn Errors of Metabolism (SSIEM)
Jul 15, 2019: Synlogic reports positive top-line data from phase 1/2a study of SYNB1618 in patients with phenylketonuria and guides to next phase of development
May 06, 2019: European Commission approves Palynziq (pegvaliase injection) for treatment of phenylketonuria (PKU) in patients aged 16 years or older
Apr 30, 2019: Synlogic presents data describing a Solid Oral Formulation Process for Synthetic Biotic Medicine SYNB1618 for the treatment of PKU at the 22nd Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT)
Apr 15, 2019: Synlogic to present data from new solid oral formulation process at the 22nd Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT)
Mar 01, 2019: BioMarin Receives Positive CHMP Opinion in Europe for Palynziq (pegvaliase Injection) for Treatment of Patients with Phenylketonuria (PKU) Aged 16 and Older
Jan 07, 2019: Retrophin provides update on phenylketonuria drug CNSA-001
Jan 07, 2019: BioMarin highlights Palynziq at 37th Annual J.P. Morgan Healthcare Conference in San Francisco
Jan 07, 2019: Homology Medicines advances first gene editing development candidate into IND-enabling studies for pediatric patients with phenylketonuria
Jan 03, 2019: Synlogic provides update on synthetic biotic medicine SYNB-1618
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer

LIST OF TABLES

Number of Products under Development for Phenylketonuria (PKU), 2021
Number of Products under Development by Companies, 2021
Products under Development by Companies, 2021
Products under Development by Companies, 2021 (Contd..1), 2021
Number of Products by Stage and Target, 2021
Number of Products by Stage and Mechanism of Action, 2021
Number of Products by Stage and Route of Administration, 2021
Number of Products by Stage and Molecule Type, 2021
Phenylketonuria (PKU) - Pipeline by Agios Pharmaceuticals Inc, 2021
Phenylketonuria (PKU) - Pipeline by American Gene Technologies International Inc, 2021
Phenylketonuria (PKU) - Pipeline by APR Applied Pharma Research SA, 2021
Phenylketonuria (PKU) - Pipeline by BioMarin Pharmaceutical Inc, 2021
Phenylketonuria (PKU) - Pipeline by Evox Therapeutics Ltd, 2021
Phenylketonuria (PKU) - Pipeline by Generation Bio Co, 2021
Phenylketonuria (PKU) - Pipeline by Homology Medicines Inc, 2021
Phenylketonuria (PKU) - Pipeline by MipSalus ApS, 2021
Phenylketonuria (PKU) - Pipeline by Moderna Inc, 2021
Phenylketonuria (PKU) - Pipeline by Nestle Health Science SA, 2021
Phenylketonuria (PKU) - Pipeline by Peg-Bio BioPharma Co Ltd, 2021
Phenylketonuria (PKU) - Pipeline by Pluvia AS, 2021
Phenylketonuria (PKU) - Pipeline by Prosit Sole Biotechnology (Beijing) Co Ltd, 2021
Phenylketonuria (PKU) - Pipeline by PTC Therapeutics Inc, 2021
Phenylketonuria (PKU) - Pipeline by Sangamo Therapeutics Inc, 2021
Phenylketonuria (PKU) - Pipeline by SOM Biotech SL, 2021
Phenylketonuria (PKU) - Pipeline by Synlogic Inc, 2021
Phenylketonuria (PKU) - Pipeline by Ultragenyx Pharmaceutical Inc, 2021
Phenylketonuria (PKU) - Pipeline by Vera Therapeutics Inc, 2021
Phenylketonuria (PKU) - Dormant Projects, 2021
Phenylketonuria (PKU) - Discontinued Products, 2021

LIST OF FIGURES

Number of Products under Development for Phenylketonuria (PKU), 2021
Number of Products under Development by Companies, 2021
Number of Products by Top 10 Targets, 2021
Number of Products by Stage and Top 10 Targets, 2021
Number of Products by Top 10 Mechanism of Actions, 2021
Number of Products by Stage and Top 10 Mechanism of Actions, 2021
Number of Products by Top 10 Routes of Administration, 2021
Number of Products by Stage and Top 10 Routes of Administration, 2021
Number of Products by Top 10 Molecule Types, 2021
Number of Products by Stage and Top 10 Molecule Types, 2021


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