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The publisher's latest Pharmaceutical and Healthcare disease pipeline guide Phenylketonuria - Drugs in Development, 2021, provides an overview of the Phenylketonuria (Metabolic Disorders) pipeline landscape.Speak directly to the analyst to clarify any post sales queries you may have.
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
The publisher'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 the publisher’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.
Table of Contents
Introduction
Phenylketonuria (PKU) - Therapeutics Development
Phenylketonuria (PKU) - Therapeutics Assessment
Phenylketonuria (PKU) - Companies Involved in Therapeutics Development
AGT-324 - Drug Profile
APROD-031 - Drug Profile
Biologic for Phenylketonuria - Drug Profile
Biologics for Phenylketonuria - Drug Profile
BMN-307 - Drug Profile
CDX-6114 - Drug Profile
Gene Therapy for Phenylketonuria and Progressive Familial Intrahepatic Cholestasis - Drug Profile
HMI-102 - Drug Profile
HMI-103 - Drug Profile
mRNA-3283 - Drug Profile
Oligonucleotides to Activate PAH for Phenylketonuria - Drug Profile
pegvaliase - Drug Profile
Phelimin - Drug Profile
PJ-008 - Drug Profile
PSP-007 - Drug Profile
PTC-923 - Drug Profile
Small Molecules to Activate Phenylalanine Hydroxylase for Phenylketonuria - Drug Profile
Small Molecules to Target PAH for Phenylketonuria - Drug Profile
SOM-1311 - Drug Profile
ST-101 - Drug Profile
SYNB-1618 - Drug Profile
UX-501 - Drug Profile
Phenylketonuria (PKU) - Dormant Projects
Phenylketonuria (PKU) - Product Development Milestones
Appendix
List of Tables
List of Figures
Companies Mentioned (Partial List)
A selection of companies mentioned in this report includes, but is not limited to:
- 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