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Ornithine-Transcarbamylase Deficiency - Pipeline Review, H2 2016

Published By :

Global Markets Direct

Published Date : Dec 2016

Category :

Pharmaceutical

No. of Pages : 37 Pages

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under Development by Companies /Universities /Institutes, the molecules developed by Companies in Filing rejected/Withdrawn, Phase III, IND/CTA Filed and Preclinical stages are 1, 1, 1 and 3 respectively.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 Directs 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 its 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

List of Figures
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 Products, H2 2016 10
Assessment by Monotherapy Products, H2 2016 17
Number of Products by Stage and Targets, H2 2016 18
Number of Products by Mechanism of Actions, H2 2016 19
Number of Products by Stage and Mechanism of Actions, H2 2016 19
Number of Products by Stage and Routes of Administration, H2 2016 21
Number of Products by Molecule Types, H2 2016 22
Number of Products by Stage and Molecule Types, H2 2016 22

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