Wilson Disease - Pipeline Review, H1 2020
Wilson Disease - Pipeline Review, H1 2020
SUMMARY
Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Wilson Disease - Pipeline Review, H1 2020, provides an overview of the Wilson Disease (Genetic Disorders) pipeline landscape.
Wilson disease is a rare autosomal recessive inherited disorder of copper metabolism that is characterized by excessive deposition of copper in the liver, brain, and other tissues. Symptoms include abdominal pain, jaundice, problems with speech, swallowing and muscle stiffness. Treatment includes chelators and Vitamin E supplements.
REPORT HIGHLIGHTS
Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Wilson Disease - Pipeline Review, H1 2020, provides comprehensive information on the therapeutics under development for Wilson Disease (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 Wilson Disease (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Wilson Disease and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies/Universities/Institutes, the molecules developed by Companies in Pre-Registration, Phase III, Preclinical and Discovery stages are 1, 2, 5 and 3 respectively. Similarly, the Universities portfolio in Preclinical stages comprises 1 molecules, respectively.
Wilson Disease (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
SUMMARY
Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Wilson Disease - Pipeline Review, H1 2020, provides an overview of the Wilson Disease (Genetic Disorders) pipeline landscape.
Wilson disease is a rare autosomal recessive inherited disorder of copper metabolism that is characterized by excessive deposition of copper in the liver, brain, and other tissues. Symptoms include abdominal pain, jaundice, problems with speech, swallowing and muscle stiffness. Treatment includes chelators and Vitamin E supplements.
REPORT HIGHLIGHTS
Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Wilson Disease - Pipeline Review, H1 2020, provides comprehensive information on the therapeutics under development for Wilson Disease (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 Wilson Disease (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Wilson Disease and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies/Universities/Institutes, the molecules developed by Companies in Pre-Registration, Phase III, Preclinical and Discovery stages are 1, 2, 5 and 3 respectively. Similarly, the Universities portfolio in Preclinical stages comprises 1 molecules, respectively.
Wilson Disease (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 Wilson Disease (Genetic Disorders).
- The pipeline guide reviews pipeline therapeutics for Wilson Disease (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 Wilson Disease (Genetic Disorders) therapeutics and enlists all their major and minor projects.
- The pipeline guide evaluates Wilson Disease (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 Wilson Disease (Genetic Disorders)
- 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 Wilson Disease (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 Wilson Disease (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.
Introduction
Global Markets Direct Report Coverage
Wilson Disease - Overview
Wilson Disease - Therapeutics Development
Pipeline Overview
Pipeline by Companies
Pipeline by Universities/Institutes
Products under Development by Companies
Products under Development by Universities/Institutes
Wilson Disease - Therapeutics Assessment
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Wilson Disease - Companies Involved in Therapeutics Development
Alexion Pharmaceuticals Inc
Aligen Therapeutics SL
Deep Genomics Inc
DepYmed Inc
Generation Bio Co
GMP-Orphan SA
Krisani Bio Sciences Pvt Ltd
LambdaGen Therapeutics
Nobelpharma Co Ltd
Ultragenyx Pharmaceutical Inc
Vivet Therapeutics SAS
Wilson Disease - Drug Profiles
CM-1186 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
DG-12P1 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
DPM-1001 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy for Wilson’s Disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate ATP7B for Wilson Disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
KBHD-001 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
methanobactin - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
tiomolibdate choline - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
trientine tetrahydrochloride - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
UX-701 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
VTX-801 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
zinc acetate - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Wilson Disease - Dormant Projects
Wilson Disease - Product Development Milestones
Featured News & Press Releases
Apr 23, 2019: Vivet Therapeutics to present on its wilson disease drug candidate VTX-801 at 2019 American Society of Gene and Cell Therapy Annual Meeting
Jun 27, 2018: Progress toward improved Wilson's disease drug
Jun 01, 2018: Wilson Therapeutics to present data from WTX101 Phase 2 extension study at 4th Congress of the European Academy of Neurology
Apr 12, 2018: Promising preliminary long-term data for WTX101 in Wilson Disease presented at EASL Annual Meeting
Mar 28, 2018: Preliminary Long-Term Data For WTX101 In Wilson Disease Accepted As A Late-Breaker Presentation At EASL Annual Meeting
Feb 16, 2018: First patient enrolled in pivotal Phase 3 FOCuS trial evaluating WTX101 for the treatment of Wilson Disease
Dec 14, 2017: WTX101 Granted Fast Track Designation by the U.S. FDA for the Treatment of Wilson Disease
Oct 23, 2017: Wilson Therapeutics reaches agreement with the FDA and EMA to initiate pivotal Phase 3 FOCuS study with WTX101 in Wilson Disease
Oct 20, 2017: Promising Preliminary Long-term Data For WTX101 In Wilson Disease Highlighted at The Liver Meeting
Oct 06, 2017: Phase 2 Clinical Trial Data for WTX101 Published in The Lancet Gastroenterology & Hepatology
Oct 01, 2017: Promising Preliminary Long-term Data from WTX101 Phase 2 Extension Study to be Presented at AASLD Annual Meeting
Sep 26, 2017: Vivet’s First Gene Therapy Product, VTX 801 for Wilson’s Disease, Receives European and US Orphan Drug Designation
Jun 08, 2017: Wilson Therapeutics presents Phase 2 data for WTX101 at MDS meeting
Apr 25, 2017: Wilson Therapeutics presents promising neurological Phase 2 data for WTX101 at AAN meeting
Apr 24, 2017: Wilson Therapeutics presented positive final Phase 2 data for WTX101 at EASL Annual Meeting
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer
Global Markets Direct Report Coverage
Wilson Disease - Overview
Wilson Disease - Therapeutics Development
Pipeline Overview
Pipeline by Companies
Pipeline by Universities/Institutes
Products under Development by Companies
Products under Development by Universities/Institutes
Wilson Disease - Therapeutics Assessment
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Wilson Disease - Companies Involved in Therapeutics Development
Alexion Pharmaceuticals Inc
Aligen Therapeutics SL
Deep Genomics Inc
DepYmed Inc
Generation Bio Co
GMP-Orphan SA
Krisani Bio Sciences Pvt Ltd
LambdaGen Therapeutics
Nobelpharma Co Ltd
Ultragenyx Pharmaceutical Inc
Vivet Therapeutics SAS
Wilson Disease - Drug Profiles
CM-1186 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
DG-12P1 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
DPM-1001 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy for Wilson’s Disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate ATP7B for Wilson Disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
KBHD-001 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
methanobactin - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
tiomolibdate choline - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
trientine tetrahydrochloride - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
UX-701 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
VTX-801 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
zinc acetate - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Wilson Disease - Dormant Projects
Wilson Disease - Product Development Milestones
Featured News & Press Releases
Apr 23, 2019: Vivet Therapeutics to present on its wilson disease drug candidate VTX-801 at 2019 American Society of Gene and Cell Therapy Annual Meeting
Jun 27, 2018: Progress toward improved Wilson's disease drug
Jun 01, 2018: Wilson Therapeutics to present data from WTX101 Phase 2 extension study at 4th Congress of the European Academy of Neurology
Apr 12, 2018: Promising preliminary long-term data for WTX101 in Wilson Disease presented at EASL Annual Meeting
Mar 28, 2018: Preliminary Long-Term Data For WTX101 In Wilson Disease Accepted As A Late-Breaker Presentation At EASL Annual Meeting
Feb 16, 2018: First patient enrolled in pivotal Phase 3 FOCuS trial evaluating WTX101 for the treatment of Wilson Disease
Dec 14, 2017: WTX101 Granted Fast Track Designation by the U.S. FDA for the Treatment of Wilson Disease
Oct 23, 2017: Wilson Therapeutics reaches agreement with the FDA and EMA to initiate pivotal Phase 3 FOCuS study with WTX101 in Wilson Disease
Oct 20, 2017: Promising Preliminary Long-term Data For WTX101 In Wilson Disease Highlighted at The Liver Meeting
Oct 06, 2017: Phase 2 Clinical Trial Data for WTX101 Published in The Lancet Gastroenterology & Hepatology
Oct 01, 2017: Promising Preliminary Long-term Data from WTX101 Phase 2 Extension Study to be Presented at AASLD Annual Meeting
Sep 26, 2017: Vivet’s First Gene Therapy Product, VTX 801 for Wilson’s Disease, Receives European and US Orphan Drug Designation
Jun 08, 2017: Wilson Therapeutics presents Phase 2 data for WTX101 at MDS meeting
Apr 25, 2017: Wilson Therapeutics presents promising neurological Phase 2 data for WTX101 at AAN meeting
Apr 24, 2017: Wilson Therapeutics presented positive final Phase 2 data for WTX101 at EASL Annual Meeting
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer
LIST OF TABLES
Number of Products under Development for Wilson Disease, 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
Wilson Disease - Pipeline by Alexion Pharmaceuticals Inc, H1 2020
Wilson Disease - Pipeline by Aligen Therapeutics SL, H1 2020
Wilson Disease - Pipeline by Deep Genomics Inc, H1 2020
Wilson Disease - Pipeline by DepYmed Inc, H1 2020
Wilson Disease - Pipeline by Generation Bio Co, H1 2020
Wilson Disease - Pipeline by GMP-Orphan SA, H1 2020
Wilson Disease - Pipeline by Krisani Bio Sciences Pvt Ltd, H1 2020
Wilson Disease - Pipeline by LambdaGen Therapeutics, H1 2020
Wilson Disease - Pipeline by Nobelpharma Co Ltd, H1 2020
Wilson Disease - Pipeline by Ultragenyx Pharmaceutical Inc, H1 2020
Wilson Disease - Pipeline by Vivet Therapeutics SAS, H1 2020
Wilson Disease - Dormant Projects, H1 2020
Number of Products under Development for Wilson Disease, 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
Wilson Disease - Pipeline by Alexion Pharmaceuticals Inc, H1 2020
Wilson Disease - Pipeline by Aligen Therapeutics SL, H1 2020
Wilson Disease - Pipeline by Deep Genomics Inc, H1 2020
Wilson Disease - Pipeline by DepYmed Inc, H1 2020
Wilson Disease - Pipeline by Generation Bio Co, H1 2020
Wilson Disease - Pipeline by GMP-Orphan SA, H1 2020
Wilson Disease - Pipeline by Krisani Bio Sciences Pvt Ltd, H1 2020
Wilson Disease - Pipeline by LambdaGen Therapeutics, H1 2020
Wilson Disease - Pipeline by Nobelpharma Co Ltd, H1 2020
Wilson Disease - Pipeline by Ultragenyx Pharmaceutical Inc, H1 2020
Wilson Disease - Pipeline by Vivet Therapeutics SAS, H1 2020
Wilson Disease - Dormant Projects, H1 2020
LIST OF FIGURES
Number of Products under Development for Wilson Disease, H1 2020
Number of Products under Development by Companies, H1 2020
Number of Products by Top 10 Targets, H1 2020
Number of Products by Stage and Top 10 Targets, H1 2020
Number of Products by Top 10 Mechanism of Actions, 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
Alexion Pharmaceuticals Inc
Aligen Therapeutics SL
Deep Genomics Inc
DepYmed Inc
Generation Bio Co
GMP-Orphan SA
Krisani Bio Sciences Pvt Ltd
LambdaGen Therapeutics
Nobelpharma Co Ltd
Ultragenyx Pharmaceutical Inc
Vivet Therapeutics SAS
Number of Products under Development for Wilson Disease, H1 2020
Number of Products under Development by Companies, H1 2020
Number of Products by Top 10 Targets, H1 2020
Number of Products by Stage and Top 10 Targets, H1 2020
Number of Products by Top 10 Mechanism of Actions, 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
Alexion Pharmaceuticals Inc
Aligen Therapeutics SL
Deep Genomics Inc
DepYmed Inc
Generation Bio Co
GMP-Orphan SA
Krisani Bio Sciences Pvt Ltd
LambdaGen Therapeutics
Nobelpharma Co Ltd
Ultragenyx Pharmaceutical Inc
Vivet Therapeutics SAS
