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Fabry Disease Pipeline and Clinical Trials Assessment (2023) | Idorsia Pharmaceuticals, Protalix, Sanofi Genzyme, Sangamo Therapeutics, 4D Molecular Therapeutics, Resverlogix Corp, and others.

08-11-2023 02:28 PM CET | Health & Medicine

Press release from: DelveInsight Business Research

Fabry Disease Pipeline

Fabry Disease Pipeline

The report titled "Fabry Disease Pipeline Insight 2023" by DelveInsight offers extensive information on more than 18+ companies and 18+ pipeline drugs in the field of Fabry Disease. It presents detailed profiles of these drugs, encompassing both Fabry Disease clinical trials and nonclinical stage products. The Fabry Disease pipeline report also includes a thorough assessment of therapeutics based on product type, stage, route of administration, and molecule type. Additionally, it highlights the inactive pipeline products in this area.

The Fabry Disease Pipeline report provides an elaborate account of the drug, encompassing its mechanism of action, Fabry Disease clinical trials studies, Fabry Disease NDA approvals (if any), and product development endeavors such as technology advancements, collaborations pertaining to Fabry Disease licensing, mergers and acquisitions, funding, designations, and other relevant details concerning the product.

To explore more information on the latest breakthroughs in the Fabry Disease Pipeline treatment landscape of the report, click here @ Fabry Disease Pipeline Outlook- https://www.delveinsight.com/report-store/fabry-disease-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr

Key Takeaways from the Fabry Disease Pipeline Report
• Over 18+ Fabry Disease companies are evaluating 18+ Fabry Disease pipeline therapies in various stages of development, and their anticipated acceptance in the Fabry Disease market would significantly increase market revenue.
• The leading Fabry Disease Companies are working in the market include Idorsia Pharmaceuticals, Protalix, Sanofi Genzyme, Sangamo Therapeutics, 4D Molecular Therapeutics, Resverlogix Corp, AVROBIO, Freeline Therapeutics, Ozmosis Research Inc., CellGenTech, Inc., uniQure, Codexis, Canbridge, Eleva GmbH, MP6 therapeutics, Amicus therapeutics, Sigilon Therapeutics, and others
• Promising Fabry Disease Pipeline therapies in various stages of development include Recombinant human alpha galactosidase A (agalsidase beta), Venglustat (GZ402671), Agalsidase beta (GZ419828), Migalastat, Lucerastat, and others
• Venglustat (also known as ibiglustat) (Sanofi Genzyme) is an orally administered small molecule being developed for several rare diseases. It is an inhibitor of an enzyme called glucosylceramide synthase (GCS) and modifies the enzyme substrates. GCS turns its substrate, ceramide, into glucosylceramide (GL-1) during lipid metabolism, a series of biochemical reactions that degrade and generate lipids. GL-1 acts as a substrate to other enzymes and is turned into globosides, a subclass of lipids where Gb3 belongs. When venglustat inhibits GCS, it prevents the synthesis of GL-1, thereby reducing the substrate of the following reactions that lead to the formation of Gb3 and its accumulation in the absence of α-galactosidase A. Due to this mechanism of action venglustat is called a substrate reduction therapy. The molecule is developed by Sanofi Genzyme and is in the Phase III (NCT05280548; NCT05206773) stage of development for FD. The company expects to file submission by 2025+.

Fabry Disease Overview
Fabry disease is an inherited lysosomal storage disease caused by a nonfunctional or partially functional enzyme, alpha-galactosidase A (α-Gal A). Decreased activity of α-Gal A in lysosomes results in the accumulation of enzyme substrates (Gb3 and lyso-Gb3) which cause cellular damage in tissues throughout the body.

For further information, refer to the detailed Fabry Disease Unmet Needs, Fabry Disease Market Drivers, and Fabry Disease Market Barriers, click here for Fabry Disease Ongoing Clinical Trial Analysis- https://www.delveinsight.com/sample-request/fabry-disease-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr

Fabry Disease Emerging Drugs Profile
• Pegunigalsidase Alfa: Protalix Biotherapeutics
• Venglustat: Sanofi
• 4D 310: 4D Molecular Therapeutics

Fabry Disease Pipeline Therapeutics Assessment
There are approx. 18+ key companies which are developing the therapies for Fabry Disease. The companies which have their Fabry Disease drug candidates in the most advanced stage, i.e. Preregistration include Protalix Biotherapeutics.

Request a sample and discover the recent advances in Fabry Disease Ongoing Clinical Trial Analysis and Medications, click here @ Fabry Disease Treatment Landscape- https://www.delveinsight.com/sample-request/fabry-disease-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr

Some of the companies in the Fabry Disease Therapeutics Market include-
Idorsia Pharmaceuticals, Protalix, Sanofi Genzyme, Sangamo Therapeutics, 4D Molecular Therapeutics, Resverlogix Corp, AVROBIO, Freeline Therapeutics, Ozmosis Research Inc., CellGenTech, Inc., uniQure, Codexis, Canbridge, Eleva GmbH, MP6 therapeutics, Amicus therapeutics, Sigilon Therapeutics, and others

Fabry Disease Pipeline Therapeutics Assessment
• Fabry Disease Assessment by Product Type
• Fabry Disease by Stage and Product Type
• Fabry Disease Assessment by Route of Administration
• Fabry Disease by Stage and Route of Administration
• Fabry Disease Assessment by Molecule Type
• Fabry Disease by Stage and Molecule Type

Dive deep into rich insights for drugs for Fabry Disease Pipeline, click here @ Fabry Disease Unmet Needs and Analyst Views- https://www.delveinsight.com/sample-request/fabry-disease-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr

Scope of the Fabry Disease Pipeline Report
• Coverage- Global
• Fabry Disease Companies- Idorsia Pharmaceuticals, Protalix, Sanofi Genzyme, Sangamo Therapeutics, 4D Molecular Therapeutics, Resverlogix Corp, AVROBIO, Freeline Therapeutics, Ozmosis Research Inc., CellGenTech, Inc., uniQure, Codexis, Canbridge, Eleva GmbH, MP6 therapeutics, Amicus therapeutics, Sigilon Therapeutics, and others
• Fabry Disease Pipeline Therapies- Recombinant human alpha galactosidase A (agalsidase beta), Venglustat (GZ402671), Agalsidase beta (GZ419828), Migalastat, Lucerastat, and others.
• Fabry Disease Pipeline Segmentation: Product Type, Molecule Type, Route of Administration

Got Queries? Find out the related information on Fabry Disease Mergers and acquisitions, Fabry Disease Licensing Activities @ Fabry Disease Emerging Drugs, and Recent Trends- https://www.delveinsight.com/sample-request/fabry-disease-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr

Table of Content
1. Introduction
2. Executive Summary
3. Fabry Disease: Overview
4. Pipeline Therapeutics
5. Therapeutic Assessment
6. Fabry Disease - DelveInsight's Analytical Perspective
7. In-depth Commercial Assessment
8. Fabry Disease Collaboration Deals
9. Late Stage Products (Preregistration)
10. Pegunigalsidase Alfa: Protalix Biotherapeutics
11. Drug profiles in the detailed report…..
12. Mid Stage Products (Phase II)
13. Venglustat: Sanofi
14. Drug profiles in the detailed report…..
15. Early stage products (Phase I/II)
16. 4D 310: 4D Molecular Therapeutics
17. Drug profiles in the detailed report…..
18. Inactive Products
19. Fabry Disease Key Companies
20. Fabry Disease Key Products
21. Fabry Disease- Unmet Needs
22. Fabry Disease- Market Drivers and Barriers
23. Fabry Disease- Future Perspectives and Conclusion
24. Fabry Disease Analyst Views
25. Fabry Disease Key Companies
26. Appendix

About Us

DelveInsight is a leading healthcare focussed market research and consulting firm that provides clients with high-quality market intelligence and analysis to support informed business decisions. With a team of experienced industry experts and a deep understanding of the life sciences and healthcare sectors, we offer customized research solutions and insights to clients across the globe. Connect with us to get high-quality, accurate, and real-time intelligence to stay ahead of the growth curve.

Company Name: DelveInsight Business Research LLP
Contact Person: Yash Bhardwaj
Email: ybhardwaj@delveinsight.com
Phone: 9193216187
Address: 304 S. Jones Blvd #2432
City: Las Vegas
State: NV
Country: United States

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