Ornithine-Transcarbamylase Deficiency – Drugs in Development, 2021

Global Markets Direct’s, ‘Ornithine-Transcarbamylase Deficiency – Drugs in Development, 2021’, provides an overview of the Ornithine-Transcarbamylase Deficiency pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Ornithine-Transcarbamylase Deficiency, complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Ornithine-Transcarbamylase Deficiency and features dormant and discontinued projects.

Global Markets Direct’s report features investigational drugs from across globe covering over 20 therapy areas and nearly 3,000 indications. The report 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. Drug profiles featured in the report undergoes periodic review following a stringent set of processes to ensure that all the profiles are updated with the latest set of information. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

The report 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.

Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data.

Scope

– The report provides a snapshot of the global therapeutic landscape of Ornithine-Transcarbamylase Deficiency

– The report reviews pipeline therapeutics for Ornithine-Transcarbamylase Deficiency by companies and universities/research institutes based on information derived from company and industry-specific sources

– The report covers pipeline products based on various stages of development ranging from pre-registration till discovery and undisclosed stages

– The report features descriptive drug profiles for the pipeline products which includes, product description, descriptive MoA, R&D brief, licensing and collaboration details & other developmental activities

– The report reviews key players involved Ornithine-Transcarbamylase Deficiency therapeutics and enlists all their major and minor projects

– The report assesses Ornithine-Transcarbamylase Deficiency therapeutics based on drug target, mechanism of action (MoA), route of administration (RoA) and molecule type

– The report summarizes all the dormant and discontinued pipeline projects

– The report reviews latest news related to pipeline therapeutics for Ornithine-Transcarbamylase Deficiency

Reasons to buy

- Gain strategically significant competitor information, analysis, and insights to formulate effective R&D strategies

- Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage

- Identify and understand important and diverse types of therapeutics under development for Ornithine-Transcarbamylase Deficiency

- Identify potential new clients or partners in the target demographic

- Develop strategic initiatives by understanding the focus areas of leading companies

- Plan mergers and acquisitions effectively by identifying key players and it’s most promising pipeline therapeutics

- Devise corrective measures for pipeline projects by understanding Ornithine-Transcarbamylase Deficiency 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

- Modify the therapeutic portfolio by identifying discontinued projects and understanding the factors that drove them from pipeline

Companies mentioned

Arcturus Therapeutics Holdings Inc

CAMP4 Therapeutics Corp

Gene Therapy Research Institution Co Ltd

Lucane Pharma SA

Poseida Therapeutics Inc

Promethera Biosciences SA

Sana Biotechnology Inc

Selecta Biosciences Inc

Ultragenyx Pharmaceutical Inc

Unicyte AG

Table of Contents

Table of Contents

List of Tables

List of Figures

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

CAMP4 Therapeutics Corp

Gene Therapy Research Institution Co Ltd

Lucane Pharma SA

Poseida Therapeutics Inc

Promethera Biosciences SA

Sana Biotechnology Inc

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

avalotcagene ontaparvovec – 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

GT-0008X – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

HepaStem – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Oligonucleotide to Activate Ornithine Transcarbamylase for Ornithine Transcarbamylase Deficiency – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Oligonucleotides for Ornithine Transcarbamylase Deficiency – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

POTC-101 – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

SEL-313 – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

SG-328 – 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, Urea Cycle Disorders and Non-Alcoholic Steatohepatitis – 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

Jul 28, 2021: Arcturus announces regulatory approval to proceed with phase 2 study of ARCT-810 mRNA therapeutic candidate for Ornithine Transcarbamylase (OTC) Deficiency

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

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, 2021

Number of Products under Development by Companies, 2021

Number of Products under Development by Universities/Institutes, 2021

Products under Development by Companies, 2021

Products under Development by Universities/Institutes, 2021

Number of Products by Stage and Target, 2021

Number of Products by Stage and Mechanism of Action, 2021

Number of Products by Stage and Route of Administration, 2021

Number of Products by Stage and Molecule Type, 2021

Ornithine-Transcarbamylase Deficiency – Pipeline by Arcturus Therapeutics Holdings Inc, 2021

Ornithine-Transcarbamylase Deficiency – Pipeline by CAMP4 Therapeutics Corp, 2021

Ornithine-Transcarbamylase Deficiency – Pipeline by Gene Therapy Research Institution Co Ltd, 2021

Ornithine-Transcarbamylase Deficiency – Pipeline by Lucane Pharma SA, 2021

Ornithine-Transcarbamylase Deficiency – Pipeline by Poseida Therapeutics Inc, 2021

Ornithine-Transcarbamylase Deficiency – Pipeline by Promethera Biosciences SA, 2021

Ornithine-Transcarbamylase Deficiency – Pipeline by Sana Biotechnology Inc, 2021

Ornithine-Transcarbamylase Deficiency – Pipeline by Selecta Biosciences Inc, 2021

Ornithine-Transcarbamylase Deficiency – Pipeline by Ultragenyx Pharmaceutical Inc, 2021

Ornithine-Transcarbamylase Deficiency – Pipeline by Unicyte AG, 2021

Ornithine-Transcarbamylase Deficiency – Dormant Projects, 2021

Ornithine-Transcarbamylase Deficiency – Discontinued Products, 2021

List of Figures

List of Figures

Number of Products under Development for Ornithine-Transcarbamylase Deficiency, 2021

Number of Products under Development by Companies, 2021

Number of Products by Stage and Targets, 2021

Number of Products by Stage and Mechanism of Actions, 2021

Number of Products by Routes of Administration, 2021

Number of Products by Stage and Routes of Administration, 2021

Number of Products by Molecule Types, 2021

Number of Products by Stage and Molecule Types, 2021

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