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Ornithine-Transcarbamylase Deficiency-Pipeline Review, H2 2017

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Ornithine-Transcarbamylase Deficiency-Pipeline Review, H2 2017

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Ornithine-Transcarbamylase Deficiency-Pipeline Review, H2 2017, provides an overview of the Ornithine-Transcarbamylase Deficiency (Genetic Disorders) pipeline landscape.

Ornithine transcarbamylase (OTC) deficiency is a rare X-linked genetic disorder characterized by complete or partial lack of the enzyme ornithine transcarbamylase (OTC). OTC plays an important role in the break down and removal of nitrogen the body (urea cycle). The lack of the OTC enzyme results in excessive accumulation of nitrogen, in the form of ammonia (hyperammonemia), in the blood. Symptoms include vomiting, refusal to eat, progressive lethargy, and coma. Treatment includes nitrogen scavenging agents.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Ornithine-Transcarbamylase Deficiency-Pipeline Review, H2 2017, provides comprehensive information on the therapeutics under development for Ornithine-Transcarbamylase Deficiency (Genetic 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 Ornithine-Transcarbamylase Deficiency (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Ornithine-Transcarbamylase Deficiency and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Filing rejected/Withdrawn, Phase III, Phase II and Preclinical stages are 1, 1, 1 and 6 respectively. Similarly, the Universities portfolio in Preclinical stages comprises 1 molecules, respectively.

Ornithine-Transcarbamylase Deficiency (Genetic 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'sproprietary 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 Ornithine-Transcarbamylase Deficiency (Genetic Disorders).

The pipeline guide reviews pipeline therapeutics for Ornithine-Transcarbamylase Deficiency (Genetic 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 Ornithine-Transcarbamylase Deficiency (Genetic Disorders) therapeutics and enlists all their major and minor projects.

The pipeline guide evaluates Ornithine-Transcarbamylase Deficiency (Genetic 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 Ornithine-Transcarbamylase Deficiency (Genetic 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 Ornithine-Transcarbamylase Deficiency (Genetic 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'smost promising pipeline therapeutics.

Formulate corrective measures for pipeline projects by understanding Ornithine-Transcarbamylase Deficiency (Genetic 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.

READ MORE

Table Of Content

Scope

Table of Contents

List of Tables

List of Figures

Introduction

Global Markets Direct Report Coverage

Ornithine-Transcarbamylase Deficiency-Overview

Ornithine-Transcarbamylase Deficiency-Therapeutics Development

Pipeline Overview

Pipeline by Companies

Pipeline by Universities/Institutes

Products under Development by Companies

Products under Development by Universities/Institutes

Ornithine-Transcarbamylase Deficiency-Therapeutics Assessment

Assessment by Target

Assessment by Mechanism of Action

Assessment by Route of Administration

Assessment by Molecule Type

Ornithine-Transcarbamylase Deficiency-Companies Involved in Therapeutics Development

Arcturus Therapeutics Inc (Inactive)

Lucane Pharma SA

PhaseRx Inc

Promethera Biosciences SA

Selecta Biosciences Inc

Translate Bio Inc

Ultragenyx Pharmaceutical Inc

Unicyte AG

Ornithine-Transcarbamylase Deficiency-Drug Profiles

DTX-301-Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Gene Therapy to Activate Ornithine Transcarbamylase for Ornithine Transcarbamylase Deficiency-Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Heparesc-Drug Profile

Product Description

Mechanism Of Action

R&D Progress

HepaStem-Drug Profile

Product Description

Mechanism Of Action

R&D Progress

LUNAR-OTC-Drug Profile

Product Description

Mechanism Of Action

R&D Progress

PRX-OTC-Drug Profile

Product Description

Mechanism Of Action

R&D Progress

SEL-313-Drug Profile

Product Description

Mechanism Of Action

R&D Progress

SHP-641-Drug Profile

Product Description

Mechanism Of Action

R&D Progress

sodium benzoate-Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Stem Cell Therapy for Type 1 Diabetes, Urea Cycle Disorders and Acute Liver Failure-Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Ornithine-Transcarbamylase Deficiency-Dormant Projects

Ornithine-Transcarbamylase Deficiency-Product Development Milestones

Featured News & Press Releases

Oct 13, 2017: PhaseRx Announces Corporate Restructuring and Review of Strategic Alternatives

Oct 05, 2017: Selecta Biosciences Announces Upcoming Clinical and Scientific Presentations on SEL-313

May 03, 2017: PhaseRx to Present Data at the American Society of Gene & Cell Therapy 20th Annual Meeting

Apr 25, 2017: PhaseRx Receives Positive Opinion for Orphan Drug Designation for PRX-OTC from European Medicines Agency

Feb 08, 2017: PhaseRx to Present Data for PRX-OTC at the 13th Annual WORLDSymposium 2017

Nov 28, 2016: PhaseRx Receives Orphan Drug Designation from FDA for PRX-OTC for the Treatment of Ornithine Transcarbamylase Deficiency

Appendix

Methodology

Coverage

Secondary Research

Primary Research

Expert Panel Validation

Contact Us

Disclaimer


List Of Figure

List of Figures

Number of Products under Development for Ornithine-Transcarbamylase Deficiency, H2 2017

Number of Products under Development by Companies, H2 2017

Number of Products by Stage and Targets, H2 2017

Number of Products by Stage and Mechanism of Actions, H2 2017

Number of Products by Stage and Routes of Administration, H2 2017

Number of Products by Top 10 Molecule Types, H2 2017

Number of Products by Stage and Molecule Types, H2 2017


List Of Table

List of Tables

Number of Products under Development for Ornithine-Transcarbamylase Deficiency, H2 2017

Number of Products under Development by Companies, H2 2017

Number of Products under Development by Universities/Institutes, H2 2017

Products under Development by Companies, H2 2017

Products under Development by Universities/Institutes, H2 2017

Number of Products by Stage and Target, H2 2017

Number of Products by Stage and Mechanism of Action, H2 2017

Number of Products by Stage and Route of Administration, H2 2017

Number of Products by Stage and Molecule Type, H2 2017

Ornithine-Transcarbamylase Deficiency-Pipeline by Arcturus Therapeutics Inc (Inactive), H2 2017

Ornithine-Transcarbamylase Deficiency-Pipeline by Lucane Pharma SA, H2 2017

Ornithine-Transcarbamylase Deficiency-Pipeline by PhaseRx Inc, H2 2017

Ornithine-Transcarbamylase Deficiency-Pipeline by Promethera Biosciences SA, H2 2017

Ornithine-Transcarbamylase Deficiency-Pipeline by Selecta Biosciences Inc, H2 2017

Ornithine-Transcarbamylase Deficiency-Pipeline by Translate Bio Inc, H2 2017

Ornithine-Transcarbamylase Deficiency-Pipeline by Ultragenyx Pharmaceutical Inc, H2 2017

Ornithine-Transcarbamylase Deficiency-Pipeline by Unicyte AG, H2 2017

Ornithine-Transcarbamylase Deficiency-Dormant Projects, H2 2017

Licence Rights

Single User License:
Report can be used by individual purchaser only

Site License: 
Report can be shared by unlimited users within one corporate location, e.g. a regional office

Corporate User License:  
Report can be shared globally by unlimited users within the purchasing corporation e.g. all employees of a single company

Section Purchase

To know more information on Purchase by Section, please send a mail to support@kenresearch.com

Products and Companies


Companies

Arcturus Therapeutics Inc (Inactive)

Lucane Pharma SA

PhaseRx Inc

Promethera Biosciences SA

Selecta Biosciences Inc

Translate Bio Inc

Ultragenyx Pharmaceutical Inc

Unicyte AG

Ornithine-Transcarbamylase Deficiency-Pipeline Review, H2 2017

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Ornithine-Transcarbamylase Deficiency-Pipeline Review, H2 2017, provides an overview of the Ornithine-Transcarbamylase Deficiency (Genetic Disorders) pipeline landscape.

Ornithine transcarbamylase (OTC) deficiency is a rare X-linked genetic disorder characterized by complete or partial lack of the enzyme ornithine transcarbamylase (OTC). OTC plays an important role in the break down and removal of nitrogen the body (urea cycle). The lack of the OTC enzyme results in excessive accumulation of nitrogen, in the form of ammonia (hyperammonemia), in the blood. Symptoms include vomiting, refusal to eat, progressive lethargy, and coma. Treatment includes nitrogen scavenging agents.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Ornithine-Transcarbamylase Deficiency-Pipeline Review, H2 2017, provides comprehensive information on the therapeutics under development for Ornithine-Transcarbamylase Deficiency (Genetic 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 Ornithine-Transcarbamylase Deficiency (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Ornithine-Transcarbamylase Deficiency and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Filing rejected/Withdrawn, Phase III, Phase II and Preclinical stages are 1, 1, 1 and 6 respectively. Similarly, the Universities portfolio in Preclinical stages comprises 1 molecules, respectively.

Ornithine-Transcarbamylase Deficiency (Genetic 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'sproprietary 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 Ornithine-Transcarbamylase Deficiency (Genetic Disorders).

The pipeline guide reviews pipeline therapeutics for Ornithine-Transcarbamylase Deficiency (Genetic 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 Ornithine-Transcarbamylase Deficiency (Genetic Disorders) therapeutics and enlists all their major and minor projects.

The pipeline guide evaluates Ornithine-Transcarbamylase Deficiency (Genetic 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 Ornithine-Transcarbamylase Deficiency (Genetic 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 Ornithine-Transcarbamylase Deficiency (Genetic 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'smost promising pipeline therapeutics.

Formulate corrective measures for pipeline projects by understanding Ornithine-Transcarbamylase Deficiency (Genetic 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.

READ MORE

Scope

Table of Contents

List of Tables

List of Figures

Introduction

Global Markets Direct Report Coverage

Ornithine-Transcarbamylase Deficiency-Overview

Ornithine-Transcarbamylase Deficiency-Therapeutics Development

Pipeline Overview

Pipeline by Companies

Pipeline by Universities/Institutes

Products under Development by Companies

Products under Development by Universities/Institutes

Ornithine-Transcarbamylase Deficiency-Therapeutics Assessment

Assessment by Target

Assessment by Mechanism of Action

Assessment by Route of Administration

Assessment by Molecule Type

Ornithine-Transcarbamylase Deficiency-Companies Involved in Therapeutics Development

Arcturus Therapeutics Inc (Inactive)

Lucane Pharma SA

PhaseRx Inc

Promethera Biosciences SA

Selecta Biosciences Inc

Translate Bio Inc

Ultragenyx Pharmaceutical Inc

Unicyte AG

Ornithine-Transcarbamylase Deficiency-Drug Profiles

DTX-301-Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Gene Therapy to Activate Ornithine Transcarbamylase for Ornithine Transcarbamylase Deficiency-Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Heparesc-Drug Profile

Product Description

Mechanism Of Action

R&D Progress

HepaStem-Drug Profile

Product Description

Mechanism Of Action

R&D Progress

LUNAR-OTC-Drug Profile

Product Description

Mechanism Of Action

R&D Progress

PRX-OTC-Drug Profile

Product Description

Mechanism Of Action

R&D Progress

SEL-313-Drug Profile

Product Description

Mechanism Of Action

R&D Progress

SHP-641-Drug Profile

Product Description

Mechanism Of Action

R&D Progress

sodium benzoate-Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Stem Cell Therapy for Type 1 Diabetes, Urea Cycle Disorders and Acute Liver Failure-Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Ornithine-Transcarbamylase Deficiency-Dormant Projects

Ornithine-Transcarbamylase Deficiency-Product Development Milestones

Featured News & Press Releases

Oct 13, 2017: PhaseRx Announces Corporate Restructuring and Review of Strategic Alternatives

Oct 05, 2017: Selecta Biosciences Announces Upcoming Clinical and Scientific Presentations on SEL-313

May 03, 2017: PhaseRx to Present Data at the American Society of Gene & Cell Therapy 20th Annual Meeting

Apr 25, 2017: PhaseRx Receives Positive Opinion for Orphan Drug Designation for PRX-OTC from European Medicines Agency

Feb 08, 2017: PhaseRx to Present Data for PRX-OTC at the 13th Annual WORLDSymposium 2017

Nov 28, 2016: PhaseRx Receives Orphan Drug Designation from FDA for PRX-OTC for the Treatment of Ornithine Transcarbamylase Deficiency

Appendix

Methodology

Coverage

Secondary Research

Primary Research

Expert Panel Validation

Contact Us

Disclaimer


List Of Figure

List of Figures

Number of Products under Development for Ornithine-Transcarbamylase Deficiency, H2 2017

Number of Products under Development by Companies, H2 2017

Number of Products by Stage and Targets, H2 2017

Number of Products by Stage and Mechanism of Actions, H2 2017

Number of Products by Stage and Routes of Administration, H2 2017

Number of Products by Top 10 Molecule Types, H2 2017

Number of Products by Stage and Molecule Types, H2 2017


List Of Table

List of Tables

Number of Products under Development for Ornithine-Transcarbamylase Deficiency, H2 2017

Number of Products under Development by Companies, H2 2017

Number of Products under Development by Universities/Institutes, H2 2017

Products under Development by Companies, H2 2017

Products under Development by Universities/Institutes, H2 2017

Number of Products by Stage and Target, H2 2017

Number of Products by Stage and Mechanism of Action, H2 2017

Number of Products by Stage and Route of Administration, H2 2017

Number of Products by Stage and Molecule Type, H2 2017

Ornithine-Transcarbamylase Deficiency-Pipeline by Arcturus Therapeutics Inc (Inactive), H2 2017

Ornithine-Transcarbamylase Deficiency-Pipeline by Lucane Pharma SA, H2 2017

Ornithine-Transcarbamylase Deficiency-Pipeline by PhaseRx Inc, H2 2017

Ornithine-Transcarbamylase Deficiency-Pipeline by Promethera Biosciences SA, H2 2017

Ornithine-Transcarbamylase Deficiency-Pipeline by Selecta Biosciences Inc, H2 2017

Ornithine-Transcarbamylase Deficiency-Pipeline by Translate Bio Inc, H2 2017

Ornithine-Transcarbamylase Deficiency-Pipeline by Ultragenyx Pharmaceutical Inc, H2 2017

Ornithine-Transcarbamylase Deficiency-Pipeline by Unicyte AG, H2 2017

Ornithine-Transcarbamylase Deficiency-Dormant Projects, H2 2017

Single User License:
Report can be used by individual purchaser only

Site License: 
Report can be shared by unlimited users within one corporate location, e.g. a regional office

Corporate User License:  
Report can be shared globally by unlimited users within the purchasing corporation e.g. all employees of a single company

To know more information on Purchase by Section, please send a mail to support@kenresearch.com

Companies

Arcturus Therapeutics Inc (Inactive)

Lucane Pharma SA

PhaseRx Inc

Promethera Biosciences SA

Selecta Biosciences Inc

Translate Bio Inc

Ultragenyx Pharmaceutical Inc

Unicyte AG