Report Detail

Pharma & Healthcare Phenylketonuria (PKU) - Pipeline Review, H2 2019

  • RnM3784258
  • |
  • 26 September, 2019
  • |
  • Global
  • |
  • 77 Pages
  • |
  • Global Markets Direct
  • |
  • Pharma & Healthcare

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.


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

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                                                                                                                                                                                                                                                                    Get latest Market Research Reports on Phenylketonuria (PKU). Industry analysis & Market Report on Phenylketonuria (PKU) is a syndicated market report, published as Phenylketonuria (PKU) - Pipeline Review, H2 2019. It is complete Research Study and Industry Analysis of Phenylketonuria (PKU) market, to understand, Market Demand, Growth, trends analysis and Factor Influencing market.

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