Phenylketonuria (PKU) - Pipeline Review, H2 2019

Global Markets Direct’s, ‘Phenylketonuria (PKU) - Pipeline Review, H2 2019’, provides an overview of the Phenylketonuria (PKU) pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Phenylketonuria (PKU), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Phenylketonuria (PKU) and features dormant and discontinued projects.

Global Markets Direct’s report features investigational drugs from across globe covering over 20 therapy areas and nearly 3,000 indications. The report 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. Drug profiles featured in the report undergoes periodic review following a stringent set of processes to ensure that all the profiles are updated with the latest set of information. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

The report 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.

Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data.

Scope

- The report provides a snapshot of the global therapeutic landscape of Phenylketonuria (PKU)

- The report reviews pipeline therapeutics for Phenylketonuria (PKU) by companies and universities/research institutes based on information derived from company and industry-specific sources

- The report covers pipeline products based on various stages of development ranging from pre-registration till discovery and undisclosed stages

- The report features descriptive drug profiles for the pipeline products which includes, product description, descriptive MoA, R&D brief, licensing and collaboration details & other developmental activities

- The report reviews key players involved Phenylketonuria (PKU) therapeutics and enlists all their major and minor projects

- The report assesses Phenylketonuria (PKU) therapeutics based on drug target, mechanism of action (MoA), route of administration (RoA) and molecule type

- The report summarizes all the dormant and discontinued pipeline projects

- The report reviews latest news related to pipeline therapeutics for Phenylketonuria (PKU)

Reasons to buy

- Gain strategically significant competitor information, analysis, and insights to formulate effective R&D strategies

- Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage

- Identify and understand important and diverse types of therapeutics under development for Phenylketonuria (PKU)

- Identify potential new clients or partners in the target demographic

- Develop strategic initiatives by understanding the focus areas of leading companies

- Plan mergers and acquisitions effectively by identifying key players and it’s most promising pipeline therapeutics

- Devise corrective measures for pipeline projects by understanding Phenylketonuria (PKU) 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

- Modify the therapeutic portfolio by identifying discontinued projects and understanding the factors that drove them from pipeline

Companies mentioned

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 ...

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

Table of Contents

Table of Contents

List of Tables

List of Figures

Introduction

Global Markets Direct Report Coverage

Phenylketonuria (PKU) – Overview

Phenylketonuria (PKU) – Therapeutics Development

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

Contact Us

Disclaimer

List of Tables

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 ...

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

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 ...

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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