Fabry Disease Pipeline: Emerging Therapies and Key pharma players involved by DelveInsight | Protalix Biotherapeutics, Sanofi, 4D Molecular therapeutics, Ozmosis Research, AvroBio and Others
Fabry Disease is a rare genetic disorder that prevents the body from making an enzyme called alpha-galactosidase A. This enzyme is responsible for breaking down a type of fat called globotriaosylceramide (Gb3 or GL-3) into building blocks that the body’s cells can use. Fabry Disease can have many symptoms because it affects cells throughout the body. The symptoms include: Chronic pain — usually a burning or tingling sensation — in the hands and feet, Clusters of small, dark red spots in various locations on the skin, Opaque corneas, resulting in cloudy-looking eyes and problems with eyesight, Ringing in the ears, and hearing loss. Fabry Disease is caused by a mutation of the GLA gene, which encodes for the alpha-galactosidase A enzyme. The mutation usually makes the enzyme unable to function, although in mild cases, it may function to some degree. The disease is inherited in an X-linked dominant manner, which means that the gene involved is situated on the X chromosome.
DelveInsight’s, “Fabry Disease Pipeline Insight, 2021,” report provides comprehensive insights about 15+ companies and 15+ pipeline drugs in Fabry Disease pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
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Some of Fabry Disease Companies are:
- Protalix Biotherapeutics
- Sanofi
- 4D Molecular therapeutics
- Ozmosis Research Inc.
- AvroBio
- Freeline Therapeutics
- Sangamo Therapeutics
- Translate Bio
- Abeona Therapeutics
- AVROBIO
- Sangamo Therapeutics
- And Many Others
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DelveInsight’s Fabry Disease report covers around 15+ products under different phases of clinical development like:
- Late stage products (Phase III)
- Mid-stage products (Phase II)
- Early-stage product (Phase I) along with the details of
- Pre-clinical and Discovery stage candidates
- Discontinued & Inactive candidates
- Route of Administration
Some of Fabry Disease Drugs are:
- Venglustat
- Pegunigalsidase alfa
- 4D 310
- And Many Others
Fabry Disease Report Key Questions:
- How many companies are developing Fabry Disease drugs?
- How many Fabry Disease drugs are developed by each company?
- How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Fabry Disease?
- What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Fabry Disease therapeutics?
- What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
- What are the clinical studies going on for Fabry Disease and their status?
- What are the key designations that have been granted to the emerging drugs?
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Table of Contents:
Introduction
Executive Summary
Fabry Disease: Overview
Pipeline Therapeutics
- Comparative Analysis
Therapeutic Assessment
Fabry Disease – DelveInsight’s Analytical Perspective
In-depth Commercial Assessment
- Fabry Disease companies’ collaborations, Licensing, Acquisition -Deal Value Trends
Fabry Disease Collaboration Deals
Late Stage Products (Preregistration)
- Comparative Analysis
Pegunigalsidase alfa: Protalix Biotherapeutics
Drug profiles in the detailed report…..
Mid Stage Products (Phase II)
- Comparative Analysis
Venglustat: Sanofi
Drug profiles in the detailed report…..
Early Stage Products (Phase I/II)
- Comparative Analysis
4D 310: 4D Molecular Therapeutics
Drug profiles in the detailed report…..
Inactive Products
- Comparative Analysis
Fabry Disease Key Companies
Fabry Disease Key Products
Fabry Disease- Unmet Needs
Fabry Disease- Market Drivers and Barriers
Fabry Disease- Future Perspectives and Conclusion
Fabry Disease Analyst Views
Fabry Disease Key Companies
Appendix
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About DelveInsight
DelveInsight is a leading Business Consultant, and Market Research Firm focused exclusively on life sciences. It supports pharma companies by providing end-to-end comprehensive solutions to improve their performance.
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