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Phenylketonuria (PKU) - Pipeline Review, H2 2019

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Date: 23-Sep-2019
No. of pages: 77
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Phenylketonuria (PKU) - Pipeline Review, H2 2019

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Phenylketonuria (PKU) - Pipeline Review, H2 2019, 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) - Pipeline Review, H2 2019, 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, IND/CTA Filed, Preclinical and Discovery stages are 1, 3, 1, 1, 11 and 1 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.

Phenylketonuria (PKU) - Pipeline Review, H2 2019

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
APR Applied Pharma Research SA
BioMarin Pharmaceutical Inc
Censa Pharmaceuticals Inc
Erytech Pharma SA
Generation Bio Corp
Homology Medicines Inc
MipSalus ApS
Moderna Therapeutics Inc
Nestle Health Science SA
Rubius Therapeutics Inc
SOM Biotech SL
Synlogic Inc
Ultragenyx Pharmaceutical Inc
Phenylketonuria (PKU) - Drug Profiles
APROD-031 - 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
Cell Therapy for Phenylketonuria - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
CNSA-001 - 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
Gene Therapy to Activate Phenylalanine Hydroxylase for Phenylketonuria - 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
pegvaliase - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Phelimin - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
RTX-134 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecules 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
SOM-1311 - 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) - Product Development Milestones
Featured News & Press Releases
Jun 27, 2019: Homology Medicines announces Leadership updates
Jun 17, 2019: Homology Medicines presents data showing single dose HMI-102 resulted in long-term correction of PKU, including reduction of Phe and increased brain neurotransmitters in murine model
Jun 12, 2019: Homology Medicines begins Phase I/II study of PKU gene therapy
Jun 05, 2019: Rubius Therapeutics announces issuance of multiple U.S. patents for its Red Cell Therapeutic Programs
May 14, 2019: Homology Medicines announces FDA Fast Track Designation for HMI-102 Gene Therapy Development Candidate for adults with PKU
May 06, 2019: European Commission approves Palynziq (pegvaliase injection) for treatment of phenylketonuria (PKU) in patients aged 16 years or older
Apr 15, 2019: Homology Medicines announces upcoming presentations on its Gene Therapy and Gene Editing platform and manufacturing capabilities at the American Society of Gene & Cell Therapy Meeting
Apr 04, 2019: Homology Medicines announces FDA clearance of Investigational New Drug Application for HMI-102 gene therapy candidate for PKU
Apr 04, 2019: Homology Medicines presents preclinical data from PKU Gene Therapy Program demonstrating restored metabolic pathway and increased neurotransmitter production
Mar 11, 2019: Rubius Therapeutics Announces FDA clearance of Investigational New Drug Application for first-ever red cell therapeutic, RTX-134, for treatment of Phenylketonuria
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: Rubius Therapeutics provides update on its phenylketonuria drug candidate RTX-134 at the 37th Annual J.P. Morgan Healthcare Conference
Jan 07, 2019: Homology Medicines achieves 2018 goals and prepares PKU Gene Therapy Program for the clinic with initial data expected in 2019
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer

List of Tables
Number of Products under Development for Phenylketonuria (PKU), H2 2019
Number of Products under Development by Companies, H2 2019
Products under Development by Companies, H2 2019
Number of Products by Stage and Target, H2 2019
Number of Products by Stage and Mechanism of Action, H2 2019
Number of Products by Stage and Route of Administration, H2 2019
Number of Products by Stage and Molecule Type, H2 2019
Phenylketonuria (PKU) - Pipeline by Agios Pharmaceuticals Inc, H2 2019
Phenylketonuria (PKU) - Pipeline by American Gene Technologies International Inc, H2 2019
Phenylketonuria (PKU) - Pipeline by APR Applied Pharma Research SA, H2 2019
Phenylketonuria (PKU) - Pipeline by BioMarin Pharmaceutical Inc, H2 2019
Phenylketonuria (PKU) - Pipeline by Censa Pharmaceuticals Inc, H2 2019
Phenylketonuria (PKU) - Pipeline by Erytech Pharma SA, H2 2019
Phenylketonuria (PKU) - Pipeline by Generation Bio Corp, H2 2019
Phenylketonuria (PKU) - Pipeline by Homology Medicines Inc, H2 2019
Phenylketonuria (PKU) - Pipeline by MipSalus ApS, H2 2019
Phenylketonuria (PKU) - Pipeline by Moderna Therapeutics Inc, H2 2019
Phenylketonuria (PKU) - Pipeline by Nestle Health Science SA, H2 2019
Phenylketonuria (PKU) - Pipeline by Rubius Therapeutics Inc, H2 2019
Phenylketonuria (PKU) - Pipeline by SOM Biotech SL, H2 2019
Phenylketonuria (PKU) - Pipeline by Synlogic Inc, H2 2019
Phenylketonuria (PKU) - Pipeline by Ultragenyx Pharmaceutical Inc, H2 2019
Phenylketonuria (PKU) - Dormant Projects, H2 2019

List of Figures
Number of Products under Development for Phenylketonuria (PKU), H2 2019
Number of Products under Development by Companies, H2 2019
Number of Products by Targets, H2 2019
Number of Products by Stage and Targets, H2 2019
Number of Products by Mechanism of Actions, H2 2019
Number of Products by Stage and Mechanism of Actions, H2 2019
Number of Products by Routes of Administration, H2 2019
Number of Products by Stage and Routes of Administration, H2 2019
Number of Products by Molecule Types, H2 2019
Number of Products by Stage and Molecule Types, H2 2019
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