Ornithine-Transcarbamylase Deficiency – Pipeline Review, H2 2017

Global Markets Direct’s, ‘Ornithine-Transcarbamylase Deficiency – Pipeline Review, H2 2017’, provides an overview of the Ornithine-Transcarbamylase Deficiency pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Ornithine-Transcarbamylase Deficiency, 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 Ornithine-Transcarbamylase Deficiency 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 Ornithine-Transcarbamylase Deficiency

The report reviews pipeline therapeutics for Ornithine-Transcarbamylase Deficiency 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 Ornithine-Transcarbamylase Deficiency therapeutics and enlists all their major and minor projects

The report assesses Ornithine-Transcarbamylase Deficiency 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 Ornithine-Transcarbamylase Deficiency

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 Ornithine-Transcarbamylase Deficiency

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 Ornithine-Transcarbamylase Deficiency 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

Arcturus Therapeutics Inc (Inactive)

Lucane Pharma SA

PhaseRx Inc

Promethera Biosciences SA

Selecta Biosciences Inc

Translate Bio Inc

Ultragenyx Pharmaceutical Inc

Unicyte AG

Table of Contents

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 Tables

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

List of Figures

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

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