Ornithine-Transcarbamylase Deficiency - Pipeline Review, H1 2020

June 2020 | 53 pages | ID: OFEAE9CD704EN
Global Markets Direct

Abstracts

Ornithine-Transcarbamylase Deficiency - Pipeline Review, H1 2020

SUMMARY

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Ornithine-Transcarbamylase Deficiency - Pipeline Review, H1 2020, 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, H1 2020, 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, Phase I and Preclinical stages are 1, 1, 1 and 4 respectively. Similarly, the Universities portfolio in Preclinical stages comprises 3 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’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.

Contents

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 Ltd
Lucane Pharma SA
Promethera Biosciences SA
Selecta Biosciences Inc
Ultragenyx Pharmaceutical Inc
Unicyte AG
Ornithine-Transcarbamylase Deficiency - Drug Profiles
ARCT-810 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
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
Gene Therapy to Activate Ornithine Transcarbamylase for Ornithine Transcarbamylase Deficiency - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate Ornithine Transcarbamylase for Ornthine Transcarbamylase Deficiency - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate Ornithine Transcarbamylase for Ornthine Transcarbamylase Deficiency - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
HepaStem - 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 Acute Liver Failure, Type 1 Diabetes and Urea Cycle Disorders - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Ornithine-Transcarbamylase Deficiency - Dormant Projects
Ornithine-Transcarbamylase Deficiency - Discontinued Products
Ornithine-Transcarbamylase Deficiency - Product Development Milestones
Featured News & Press Releases
Jun 05, 2020: Arcturus Therapeutics announces first healthy volunteer dosed in phase 1 study of ARCT-810 for Ornithine Transcarbamylase (OTC) Deficiency
Apr 13, 2020: Arcturus Therapeutics announces allowance of IND & approval of clinical trial application (CTA) for ARCT-810, a first-in-class investigational mRNA medicine to treat ornithine transcarbamylase deficiency
Oct 23, 2019: Selecta Biosciences presents new preclinical data from its gene therapy program at 2019 ESGCT Annual Congress
Apr 16, 2019: Selecta Biosciences presents new preclinical data of SEL-313 from its Gene Therapy Program at the American Society of Gene & Cell Therapy (ASGCT) 22nd Annual Meeting
Oct 05, 2018: Co-administration of AAV Vectors with SVP-Rapamycin Enables Vector Re-administration in Pre-clinical Gene Therapy Study Published in Nature Communications by Genethon and Selecta Biosciences
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

LIST OF TABLES

Number of Products under Development for Ornithine-Transcarbamylase Deficiency, H1 2020
Number of Products under Development by Companies, H1 2020
Number of Products under Development by Universities/Institutes, H1 2020
Products under Development by Companies, H1 2020
Products under Development by Universities/Institutes, H1 2020
Number of Products by Stage and Target, H1 2020
Number of Products by Stage and Mechanism of Action, H1 2020
Number of Products by Stage and Route of Administration, H1 2020
Number of Products by Stage and Molecule Type, H1 2020
Ornithine-Transcarbamylase Deficiency - Pipeline by Arcturus Therapeutics Ltd, H1 2020
Ornithine-Transcarbamylase Deficiency - Pipeline by Lucane Pharma SA, H1 2020
Ornithine-Transcarbamylase Deficiency - Pipeline by Promethera Biosciences SA, H1 2020
Ornithine-Transcarbamylase Deficiency - Pipeline by Selecta Biosciences Inc, H1 2020
Ornithine-Transcarbamylase Deficiency - Pipeline by Ultragenyx Pharmaceutical Inc, H1 2020
Ornithine-Transcarbamylase Deficiency - Pipeline by Unicyte AG, H1 2020
Ornithine-Transcarbamylase Deficiency - Dormant Projects, H1 2020
Ornithine-Transcarbamylase Deficiency - Discontinued Products, H1 2020

List Of Figures

LIST OF FIGURES

Number of Products under Development for Ornithine-Transcarbamylase Deficiency, H1 2020
Number of Products under Development by Companies, H1 2020
Number of Products by Stage and Top 10 Targets, H1 2020
Number of Products by Stage and Top 10 Mechanism of Actions, H1 2020
Number of Products by Stage and Top 10 Routes of Administration, H1 2020
Number of Products by Top 10 Molecule Types, H1 2020
Number of Products by Stage and Top 10 Molecule Types, H1 2020

COMPANIES MENTIONED

Arcturus Therapeutics Ltd
Lucane Pharma SA
Promethera Biosciences SA
Selecta Biosciences Inc
Ultragenyx Pharmaceutical Inc
Unicyte AG