Fabry Disease - Pipeline Review, H2 2020
Fabry Disease - Pipeline Review, H2 2020
Summary
Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Fabry Disease - Pipeline Review, H2 2020, provides an overview of the Fabry Disease (Genetic Disorders) pipeline landscape.
Fabry disease is an X-linked lysosomal disorder that results in abnormal deposits of globotriaosylceramide in blood vessel walls throughout the body. It is caused due to mutations in GLA gene. The GLA gene controls the production of a particular enzyme called alpha-galactosidase A (this enzyme is responsible for breaking down of globotriaosylceramide). Symptoms include skin rash, cramps, gas, diarrhea, heart enlargement, angina, dizziness, headache, nausea, and heat intolerance. Treatment includes enzyme replacement therapy (ERT) and pain management.
Report Highlights
Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Fabry Disease - Pipeline Review, H2 2020, provides comprehensive information on the therapeutics under development for Fabry 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 Fabry Disease (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Fabry 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, Phase II, IND/CTA Filed, Preclinical, Discovery and Unknown stages are 1, 1, 6, 1, 11, 2 and 1 respectively. Similarly, the Universities portfolio in Discovery and Unknown stages comprises 1 and 1 molecules, respectively.
Fabry 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 Fabry Disease - Pipeline Review, H2 2020, provides an overview of the Fabry Disease (Genetic Disorders) pipeline landscape.
Fabry disease is an X-linked lysosomal disorder that results in abnormal deposits of globotriaosylceramide in blood vessel walls throughout the body. It is caused due to mutations in GLA gene. The GLA gene controls the production of a particular enzyme called alpha-galactosidase A (this enzyme is responsible for breaking down of globotriaosylceramide). Symptoms include skin rash, cramps, gas, diarrhea, heart enlargement, angina, dizziness, headache, nausea, and heat intolerance. Treatment includes enzyme replacement therapy (ERT) and pain management.
Report Highlights
Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Fabry Disease - Pipeline Review, H2 2020, provides comprehensive information on the therapeutics under development for Fabry 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 Fabry Disease (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Fabry 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, Phase II, IND/CTA Filed, Preclinical, Discovery and Unknown stages are 1, 1, 6, 1, 11, 2 and 1 respectively. Similarly, the Universities portfolio in Discovery and Unknown stages comprises 1 and 1 molecules, respectively.
Fabry 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 Fabry Disease (Genetic Disorders).
- The pipeline guide reviews pipeline therapeutics for Fabry 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 Fabry Disease (Genetic Disorders) therapeutics and enlists all their major and minor projects.
- The pipeline guide evaluates Fabry 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 Fabry 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 Fabry 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 Fabry 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
Fabry Disease - Overview
Fabry Disease - Therapeutics Development
Fabry Disease - Therapeutics Assessment
Fabry Disease - Companies Involved in Therapeutics Development
Fabry Disease - Drug Profiles
Fabry Disease - Dormant Projects
Fabry Disease - Discontinued Products
Fabry Disease - Product Development Milestones
Appendix
Fabry Disease - Overview
Fabry Disease - Therapeutics Development
Fabry Disease - Therapeutics Assessment
Fabry Disease - Companies Involved in Therapeutics Development
Fabry Disease - Drug Profiles
Fabry Disease - Dormant Projects
Fabry Disease - Discontinued Products
Fabry Disease - Product Development Milestones
Appendix
LIST OF TABLES
Number of Products under Development for Fabry Disease, H2 2020
Number of Products under Development by Companies, H2 2020
Number of Products under Development by Universities/Institutes, H2 2020
Products under Development by Companies, H2 2020
Products under Development by Companies, H2 2020 (Contd..1), H2 2020
Products under Development by Universities/Institutes, H2 2020
Number of Products by Stage and Target, H2 2020
Number of Products by Stage and Mechanism of Action, H2 2020
Number of Products by Stage and Route of Administration, H2 2020
Number of Products by Stage and Molecule Type, H2 2020
Fabry Disease - Pipeline by 4D Molecular Therapeutics Inc, H2 2020
Fabry Disease - Pipeline by Abeona Therapeutics Inc, H2 2020
Fabry Disease - Pipeline by AceLink Therapeutics Inc, H2 2020
Fabry Disease - Pipeline by Amicus Therapeutics Inc, H2 2020
Fabry Disease - Dormant Projects, H2 2020
Fabry Disease - Discontinued Products, H2 2020
Number of Products under Development for Fabry Disease, H2 2020
Number of Products under Development by Companies, H2 2020
Number of Products under Development by Universities/Institutes, H2 2020
Products under Development by Companies, H2 2020
Products under Development by Companies, H2 2020 (Contd..1), H2 2020
Products under Development by Universities/Institutes, H2 2020
Number of Products by Stage and Target, H2 2020
Number of Products by Stage and Mechanism of Action, H2 2020
Number of Products by Stage and Route of Administration, H2 2020
Number of Products by Stage and Molecule Type, H2 2020
Fabry Disease - Pipeline by 4D Molecular Therapeutics Inc, H2 2020
Fabry Disease - Pipeline by Abeona Therapeutics Inc, H2 2020
Fabry Disease - Pipeline by AceLink Therapeutics Inc, H2 2020
Fabry Disease - Pipeline by Amicus Therapeutics Inc, H2 2020
Fabry Disease - Dormant Projects, H2 2020
Fabry Disease - Discontinued Products, H2 2020
LIST OF FIGURES
Number of Products under Development for Fabry Disease, H2 2020
Number of Products under Development by Companies, H2 2020
Number of Products under Development by Universities/Institutes, H2 2020
Number of Products by Targets, H2 2020
Number of Products by Stage and Targets, H2 2020
Number of Products by Mechanism of Actions, H2 2020
Number of Products by Stage and Mechanism of Actions, H2 2020
Number of Products by Routes of Administration, H2 2020
Number of Products by Stage and Routes of Administration, H2 2020
Number of Products by Molecule Types, H2 2020
Number of Products by Stage and Molecule Types, H2 2020
COMPANIES MENTIONED
4D Molecular Therapeutics Inc
Abeona Therapeutics Inc
AceLink Therapeutics Inc
Amicus Therapeutics Inc
AVROBIO Inc
Biosidus SA
Chiesi Farmaceutici SpA
eleva GmbH
Freeline Therapeutics Ltd
Genzyme Corp
Idorsia Pharmaceutical Ltd
M6P Therapeutics
Pharming Group NV
Resverlogix Corp
Sangamo Therapeutics Inc
Sigilon Therapeutics Inc
Takeda Pharmaceutical Co Ltd
UniQure NV
Yuhan Corp
Number of Products under Development for Fabry Disease, H2 2020
Number of Products under Development by Companies, H2 2020
Number of Products under Development by Universities/Institutes, H2 2020
Number of Products by Targets, H2 2020
Number of Products by Stage and Targets, H2 2020
Number of Products by Mechanism of Actions, H2 2020
Number of Products by Stage and Mechanism of Actions, H2 2020
Number of Products by Routes of Administration, H2 2020
Number of Products by Stage and Routes of Administration, H2 2020
Number of Products by Molecule Types, H2 2020
Number of Products by Stage and Molecule Types, H2 2020
COMPANIES MENTIONED
4D Molecular Therapeutics Inc
Abeona Therapeutics Inc
AceLink Therapeutics Inc
Amicus Therapeutics Inc
AVROBIO Inc
Biosidus SA
Chiesi Farmaceutici SpA
eleva GmbH
Freeline Therapeutics Ltd
Genzyme Corp
Idorsia Pharmaceutical Ltd
M6P Therapeutics
Pharming Group NV
Resverlogix Corp
Sangamo Therapeutics Inc
Sigilon Therapeutics Inc
Takeda Pharmaceutical Co Ltd
UniQure NV
Yuhan Corp
