Ornithine-Transcarbamylase Deficiency - Pipeline Review, H1 2018

  • ID: 4576075
  • Drug Pipelines
  • 38 pages
  • Global Markets Direct
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FEATURED COMPANIES

  • Arcturus Therapeutics Ltd
  • Lucane Pharma SA
  • Promethera Biosciences SA
  • Selecta Biosciences Inc
  • Translate Bio Inc
  • Unicyte AG
  • MORE
Ornithine-Transcarbamylase Deficiency - Pipeline Review, H1 2018

Summary:

This latest Pharmaceutical and Healthcare disease pipeline guide Ornithine-Transcarbamylase Deficiency - Pipeline Review, H1 2018, 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:

This latest pipeline guide Ornithine-Transcarbamylase Deficiency - Pipeline Review, H1 2018, 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 Phase III, Phase II and Preclinical stages are 1, 1 and 5 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 the researcher'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 for Purchase:
  • 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.
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Note: Product cover images may vary from those shown
2 of 4

FEATURED COMPANIES

  • Arcturus Therapeutics Ltd
  • Lucane Pharma SA
  • Promethera Biosciences SA
  • Selecta Biosciences Inc
  • Translate Bio Inc
  • Unicyte AG
  • MORE
Introduction

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 Ltd

Lucane Pharma SA

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

HepaStem - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

LUNAR-OTC - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

MRT-5201 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

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

Appendix

Methodology

Coverage

Secondary Research

Primary Research

Expert Panel Validation

Contact Us

Disclaimer

List of Tables

Number of Products under Development for Ornithine-Transcarbamylase Deficiency, H1 2018

Number of Products under Development by Companies, H1 2018

Number of Products under Development by Universities/Institutes, H1 2018

Products under Development by Companies, H1 2018

Products under Development by Universities/Institutes, H1 2018

Number of Products by Stage and Target, H1 2018

Number of Products by Stage and Mechanism of Action, H1 2018

Number of Products by Stage and Route of Administration, H1 2018

Number of Products by Stage and Molecule Type, H1 2018

Ornithine-Transcarbamylase Deficiency - Pipeline by Arcturus Therapeutics Ltd, H1 2018

Ornithine-Transcarbamylase Deficiency - Pipeline by Lucane Pharma SA, H1 2018

Ornithine-Transcarbamylase Deficiency - Pipeline by Promethera Biosciences SA, H1 2018

Ornithine-Transcarbamylase Deficiency - Pipeline by Selecta Biosciences Inc, H1 2018

Ornithine-Transcarbamylase Deficiency - Pipeline by Translate Bio Inc, H1 2018

Ornithine-Transcarbamylase Deficiency - Pipeline by Ultragenyx Pharmaceutical Inc, H1 2018

Ornithine-Transcarbamylase Deficiency - Pipeline by Unicyte AG, H1 2018

Ornithine-Transcarbamylase Deficiency - Dormant Projects, H1 2018

List of Figures

Number of Products under Development for Ornithine-Transcarbamylase Deficiency, H1 2018

Number of Products under Development by Companies, H1 2018

Number of Products by Stage and Targets, H1 2018

Number of Products by Stage and Mechanism of Actions, H1 2018

Number of Products by Stage and Routes of Administration, H1 2018

Number of Products by Molecule Types, H1 2018

Number of Products by Stage and Molecule Types, H1
Note: Product cover images may vary from those shown
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  • Arcturus Therapeutics Ltd
  • Lucane Pharma SA
  • Promethera Biosciences SA
  • Selecta Biosciences Inc
  • Translate Bio Inc
  • Ultragenyx Pharmaceutical Inc
  • Unicyte AG
Note: Product cover images may vary from those shown
5 of 4
Note: Product cover images may vary from those shown
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