Report Detail

Pharma & Healthcare Phenylketonuria (PKU) - Pipeline Review, H1 2019

  • RnM2890042
  • |
  • 06 February, 2019
  • |
  • Global
  • |
  • 67 Pages
  • |
  • Global Markets Direct
  • |
  • Pharma & Healthcare

Phenylketonuria (PKU) - Pipeline Review, H1 2019

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Phenylketonuria (PKU) - Pipeline Review, H1 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, H1 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 Pre-Registration, Phase II, Phase I, Preclinical and Discovery stages are 1, 2, 1, 12 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

                                      BioMarin Pharmaceutical Inc

                                        Censa Pharmaceuticals Inc

                                          Codexis Inc

                                            Erytech Pharma SA

                                              Generation Bio Corp

                                                Homology Medicines Inc

                                                  MipSalus ApS

                                                    Moderna Therapeutics Inc

                                                      Rubius Therapeutics Inc

                                                        Synlogic Inc

                                                          Synthetic Biologics Inc

                                                            Ultragenyx Pharmaceutical Inc

                                                              Phenylketonuria (PKU) - Drug Profiles

                                                                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

                                                                                                                                                                                SYN-200 - 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

                                                                                                                                                                                                              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

                                                                                                                                                                                                                      Jan 07, 2019: Homology Medicines advances first gene editing development candidate into IND-enabling studies for pediatric patients with phenylketonuria

                                                                                                                                                                                                                        Nov 08, 2018: Codexis announces results of phase 1a clinical trial with CDX-6114

                                                                                                                                                                                                                          Nov 07, 2018: BioMarin provides update on Palynziq at at R&D Day in New York

                                                                                                                                                                                                                            Nov 07, 2018: BioMarin provides update on BMN 307, a pre-clinical gene therapy for PKU

                                                                                                                                                                                                                              Oct 18, 2018: American Gene Technologies granted FDA orphan drug designation for phenylketonuria

                                                                                                                                                                                                                                Oct 18, 2018: Homology Medicines Presents Long-Term Efficacy Data of a Single Dose of Gene Therapy Development Candidate for Treatment of Phenylketonuria

                                                                                                                                                                                                                                  Jul 11, 2018: Codexis Appoints Dr. Hicham Alaoui, Veteran Drug Discoverer, as Vice President, Biotherapeutics Research & Development

                                                                                                                                                                                                                                    Jul 09, 2018: Codexis Doses First Subjects in Phase 1a Trial of CDX-6114

                                                                                                                                                                                                                                      Jul 05, 2018: Homology Medicines to present at the National PKU Alliance Conference

                                                                                                                                                                                                                                        May 24, 2018: BioMarin Receives Standard Approval for Palynziq (pegvaliase-pqpz) Injection for Treatment of Adults with Phenylketonuria (PKU), a Rare Genetic Disease

                                                                                                                                                                                                                                          May 18, 2018: Homology Medicines Presents Data from PKU Gene Therapy Program Demonstrating Long-Term Durability of Treatment in IND-Enabling Studies

                                                                                                                                                                                                                                            Appendix

                                                                                                                                                                                                                                              Methodology

                                                                                                                                                                                                                                                Coverage

                                                                                                                                                                                                                                                  Secondary Research

                                                                                                                                                                                                                                                    Primary Research

                                                                                                                                                                                                                                                      Expert Panel Validation

                                                                                                                                                                                                                                                        Contact Us

                                                                                                                                                                                                                                                          Disclaimer

                                                                                                                                                                                                                                                          Summary:
                                                                                                                                                                                                                                                          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, H1 2019. It is complete Research Study and Industry Analysis of Phenylketonuria (PKU) market, to understand, Market Demand, Growth, trends analysis and Factor Influencing market.

                                                                                                                                                                                                                                                          Last updated on

                                                                                                                                                                                                                                                          REPORT YOU MIGHT BE INTERESTED

                                                                                                                                                                                                                                                          Purchase this Report

                                                                                                                                                                                                                                                          $2,000.00
                                                                                                                                                                                                                                                          $4,000.00
                                                                                                                                                                                                                                                          $6,000.00
                                                                                                                                                                                                                                                          1,594.00
                                                                                                                                                                                                                                                          3,188.00
                                                                                                                                                                                                                                                          4,782.00
                                                                                                                                                                                                                                                          1,858.00
                                                                                                                                                                                                                                                          3,716.00
                                                                                                                                                                                                                                                          5,574.00
                                                                                                                                                                                                                                                          306,460.00
                                                                                                                                                                                                                                                          612,920.00
                                                                                                                                                                                                                                                          919,380.00
                                                                                                                                                                                                                                                          166,880.00
                                                                                                                                                                                                                                                          333,760.00
                                                                                                                                                                                                                                                          500,640.00
                                                                                                                                                                                                                                                          Credit card Logo

                                                                                                                                                                                                                                                          Related Reports


                                                                                                                                                                                                                                                          Reason to Buy

                                                                                                                                                                                                                                                          Request for Sample of this report