Ornithine-Transcarbamylase Deficiency Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update

November 2022 | 61 pages | ID: O81CB99C1514EN
Global Markets Direct

Abstracts

Ornithine-Transcarbamylase Deficiency Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update

SUMMARY

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Ornithine-Transcarbamylase Deficiency - Drugs In Development, 2022, 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 - Drugs In Development, 2022, 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, 3 and 9 respectively. Similarly, the Universities portfolio in Preclinical stages comprises 2 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 Holdings Inc
BioNTech SE
Bloomsbury Genetic Therapies Ltd
CAMP4 Therapeutics Corp
Cellaion SA
Gene Therapy Research Institution Co Ltd
iECURE
Lucane Pharma SA
Moderna Inc
Poseida Therapeutics Inc
Sana Biotechnology Inc
Ultragenyx Pharmaceutical Inc
Unicyte AG
Ornithine-Transcarbamylase Deficiency - Drug Profiles
ARCT-810 - Drug Profile
Product Description
Mechanism Of Action
avalotcagene ontaparvovec - Drug Profile
Product Description
Mechanism Of Action
BGT-OTCD - Drug Profile
Product Description
Mechanism Of Action
BNT-171 - Drug Profile
Product Description
Mechanism Of Action
Gene Therapy to Activate Ornithine Transcarbamylase for Ornthine Transcarbamylase Deficiency - Drug Profile
Product Description
Mechanism Of Action
GT-0008X - Drug Profile
Product Description
Mechanism Of Action
GTP-506 - Drug Profile
Product Description
Mechanism Of Action
HepaStem - Drug Profile
Product Description
Mechanism Of Action
HLSC-001 - Drug Profile
Product Description
Mechanism Of Action
mRNA-3139 - Drug Profile
Product Description
Mechanism Of Action
Oligonucleotide to Activate Ornithine Transcarbamylase for Ornithine Transcarbamylase Deficiency - Drug Profile
Product Description
Mechanism Of Action
Oligonucleotides for Ornithine Transcarbamylase Deficiency - Drug Profile
Product Description
Mechanism Of Action
POTC-101 - Drug Profile
Product Description
Mechanism Of Action
SG-328 - Drug Profile
Product Description
Mechanism Of Action
sodium benzoate - Drug Profile
Product Description
Mechanism Of Action
Ornithine-Transcarbamylase Deficiency - Dormant Projects
Ornithine-Transcarbamylase Deficiency - Discontinued Products
Ornithine-Transcarbamylase Deficiency - Product Development Milestones
Featured News & Press Releases
Oct 19, 2022: Preclinical data from iECURE’s GTP-506 demonstrates potential for the treatment of Ornithine Transcarbamylase (OTC) Deficiency
Aug 24, 2022: FDA grants Rare Pediatric Disease status for iECURE’s OTC deficiency therapy
May 19, 2022: Ultragenyx announces positive longer-term durability data from Phase 1/2 Gene Therapy Study of DTX301 at American Society of Gene & Cell Therapy (ASGCT) 2022 Annual Meeting
May 19, 2022: Ultragenyx announces upcoming data presentations at American Society of Gene & Cell Therapy (ASGCT) 2022 Annual Meeting
Nov 29, 2021: Ultragenyx announces additional positive multi-year durability data from phase 1/2 AAV gene therapy studies of DTX301
Jul 28, 2021: Arcturus announces regulatory approval to proceed with phase 2 study of ARCT-810 mRNA therapeutic candidate for Ornithine Transcarbamylase (OTC) Deficiency
May 14, 2021: Ultragenyx announces presentation on its gene therapy candidate DTX301 at American Society of Gene & Cell Therapy 2021 Annual Meeting
Apr 22, 2021: Ultragenyx completes successful end-of-phase 2 meeting with FDA and finalizes phase 3 study design for DTX301 Ornithine Transcarbamylase (OTC) gene therapy program
Dec 07, 2020: Arcturus Therapeutics announces initiation of dosing ARCT-810 in patients with Ornithine Transcarbamylase (OTC) deficiency
Oct 05, 2020: Arcturus Therapeutics announces completion of first three dose escalation cohorts in phase 1 study of ARCT-810, therapeutic candidate for ornithine transcarbamylase (OTC) deficiency
Jun 05, 2020: Arcturus Therapeutics announces first healthy volunteer dosed in phase 1 study of ARCT-810 for Ornithine Transcarbamylase (OTC) Deficiency
May 13, 2020: Ultragenyx announces positive longer-term results from first three cohorts of phase 1/2 study of DTX301 Gene Therapy in 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
Jan 09, 2020: Ultragenyx announces positive topline cohort 3 results and improved longer-term cohort 2 results from phase 1/2 study of DTX301 gene therapy in ornithine transcarbamylase (OTC) deficiency
Sep 27, 2018: Ultragenyx announces positive topline cohort 2 results from phase 1/2 clinical study of DTX301 gene therapy in ornithine transcarbamylase (OTC) deficiency and progression to higher dose
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, 2022
Number of Products under Development by Companies, 2022
Number of Products under Development by Universities/Institutes, 2022
Products under Development by Companies, 2022
Products under Development by Universities/Institutes, 2022
Number of Products by Stage and Target, 2022
Number of Products by Stage and Mechanism of Action, 2022
Number of Products by Stage and Route of Administration, 2022
Number of Products by Stage and Molecule Type, 2022
Ornithine-Transcarbamylase Deficiency - Pipeline by Arcturus Therapeutics Holdings Inc, 2022
Ornithine-Transcarbamylase Deficiency - Pipeline by BioNTech SE, 2022
Ornithine-Transcarbamylase Deficiency - Pipeline by Bloomsbury Genetic Therapies Ltd, 2022
Ornithine-Transcarbamylase Deficiency - Pipeline by CAMP4 Therapeutics Corp, 2022
Ornithine-Transcarbamylase Deficiency - Pipeline by Cellaion SA, 2022
Ornithine-Transcarbamylase Deficiency - Pipeline by Gene Therapy Research Institution Co Ltd, 2022
Ornithine-Transcarbamylase Deficiency - Pipeline by iECURE, 2022
Ornithine-Transcarbamylase Deficiency - Pipeline by Lucane Pharma SA, 2022
Ornithine-Transcarbamylase Deficiency - Pipeline by Moderna Inc, 2022
Ornithine-Transcarbamylase Deficiency - Pipeline by Poseida Therapeutics Inc, 2022
Ornithine-Transcarbamylase Deficiency - Pipeline by Sana Biotechnology Inc, 2022
Ornithine-Transcarbamylase Deficiency - Pipeline by Ultragenyx Pharmaceutical Inc, 2022
Ornithine-Transcarbamylase Deficiency - Pipeline by Unicyte AG, 2022
Ornithine-Transcarbamylase Deficiency - Dormant Projects, 2022
Ornithine-Transcarbamylase Deficiency - Discontinued Products, 2022

List Of Figures

LIST OF FIGURES

Number of Products under Development for Ornithine-Transcarbamylase Deficiency, 2022
Number of Products under Development by Companies, 2022
Number of Products by Stage and Top 10 Targets, 2022
Number of Products by Stage and Top 10 Mechanism of Actions, 2022
Number of Products by Top 10 Routes of Administration, 2022
Number of Products by Stage and Top 10 Routes of Administration, 2022
Number of Products by Top 10 Molecule Types, 2022
Number of Products by Stage and Top 10 Molecule Types, 2022