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Ornithine-Transcarbamylase Deficiency - Pipeline Review, H2 2016

Published: Dec, 2016 | Pages: 37 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

Ornithine-Transcarbamylase Deficiency - Pipeline Review, H2 2016

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Ornithine-Transcarbamylase Deficiency - Pipeline Review, H2 2016, 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 2016, 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under Development by Companies /Universities /Institutes, the molecules developed by Companies in Filing rejected/Withdrawn, Phase III, IND/CTA Filed and Preclinical stages are 1, 1, 1 and 3 respectively.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'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 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's most 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.
 Table of Contents
Table of Contents 2 List of Tables 4 List of Figures 4 Introduction 5 Global Markets Direct Report Coverage 5 Ornithine-Transcarbamylase Deficiency Overview 6 Therapeutics Development 7 Pipeline Products for Ornithine-Transcarbamylase Deficiency - Overview 7 Ornithine-Transcarbamylase Deficiency - Therapeutics under Development by Companies 8 Ornithine-Transcarbamylase Deficiency - Pipeline Products Glance 9 Late Stage Products 9 Early Stage Products 10 Ornithine-Transcarbamylase Deficiency - Products under Development by Companies 11 Ornithine-Transcarbamylase Deficiency - Companies Involved in Therapeutics Development 12 Dimension Therapeutics Inc 12 Lucane Pharma SA 13 PhaseRx Inc 14 Promethera Biosciences SA 15 Unicyte AG 16 Ornithine-Transcarbamylase Deficiency - Therapeutics Assessment 17 Assessment by Monotherapy Products 17 Assessment by Target 18 Assessment by Mechanism of Action 19 Assessment by Route of Administration 21 Assessment by Molecule Type 22 Drug Profiles 24 Cell Therapy for Urea Cycle Disorders and Liver Failure - Drug Profile 24 Product Description 24 Mechanism Of Action 24 R&D Progress 24 DTX-301 - Drug Profile 26 Product Description 26 Mechanism Of Action 26 R&D Progress 26 HepaStem - Drug Profile 28 Product Description 28 Mechanism Of Action 28 R&D Progress 28 PRX-OTC - Drug Profile 31 Product Description 31 Mechanism Of Action 31 R&D Progress 31 sodium benzoate - Drug Profile 32 Product Description 32 Mechanism Of Action 32 R&D Progress 32 Stem Cell Therapy for Type 1 Diabetes, Urea Cycle Disorders and Acute Liver Failure - Drug Profile 33 Product Description 33 Mechanism Of Action 33 R&D Progress 33 Ornithine-Transcarbamylase Deficiency - Dormant Projects 34 Ornithine-Transcarbamylase Deficiency - Product Development Milestones 35 Featured News & Press Releases 35 Nov 28, 2016: PhaseRx Receives Orphan Drug Designation from FDA for PRX-OTC for the Treatment of Ornithine Transcarbamylase Deficiency 35 Appendix 36 Methodology 36 Coverage 36 Secondary Research 36 Primary Research 36 Expert Panel Validation 36 Contact Us 36 Disclaimer 37
List of Tables
Number of Products under Development for Ornithine-Transcarbamylase Deficiency, H2 2016 7 Number of Products under Development by Companies, H2 2016 8 Comparative Analysis by Late Stage Development, H2 2016 9 Comparative Analysis by Early Stage Development, H2 2016 10 Products under Development by Companies, H2 2016 11 Ornithine-Transcarbamylase Deficiency - Pipeline by Dimension Therapeutics Inc, H2 2016 12 Ornithine-Transcarbamylase Deficiency - Pipeline by Lucane Pharma SA, H2 2016 13 Ornithine-Transcarbamylase Deficiency - Pipeline by PhaseRx Inc, H2 2016 14 Ornithine-Transcarbamylase Deficiency - Pipeline by Promethera Biosciences SA, H2 2016 15 Ornithine-Transcarbamylase Deficiency - Pipeline by Unicyte AG, H2 2016 16 Assessment by Monotherapy Products, H2 2016 17 Number of Products by Stage and Target, H2 2016 18 Number of Products by Stage and Mechanism of Action, H2 2016 20 Number of Products by Stage and Route of Administration, H2 2016 21 Number of Products by Stage and Molecule Type, H2 2016 23 Ornithine-Transcarbamylase Deficiency - Dormant Projects, H2 2016 34



                                

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