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The publisher'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.Speak directly to the analyst to clarify any post sales queries you may have.
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
The publisher'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 the publisher'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
- 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 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
- 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
- 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
Companies Mentioned (Partial List)
A selection of companies mentioned in this report includes, but is not limited to:
- 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
