Press release
Gaucher's Disease Pipeline as Novel and Extensive 16+ Therapies Likely to Enter in the Treatment Domain
Key takeaways from the Gaucher's Disease Pipeline Report
• DelveInsight's Gaucher's Disease Pipeline report depicts a robust space with 14+ active players working to develop 16+ pipeline therapies for Gaucher's Disease.
• The leading Gaucher's Disease Companies such as Gain therapeutics, M6P Therapeutics, AvroBio, Orphazyme, Eli Lilly and Company, Sanofi, and others, are developing therapies for the Gaucher's disease treatment
• Gaucher's disease Emerging therapies such as - GBA programme, M 011, AVR-RD-02, Arimoclomol, GBA1 Gene Therapy, Venglustat, and others are expected to have a significant impact on the Gaucher's disease market in the coming years.
• The Gaucher's disease companies and academics are working to assess challenges and seek opportunities that could influence Gaucher's Disease R&D. The therapies under development are focused on novel approaches to treat/improve Gaucher's Disease.
Request a sample and discover the recent advances in Gaucher's Disease Treatment Drugs Report @ https://www.delveinsight.com/sample-request/gauchers-disease-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr
Gaucher's Disease Overview
Gaucher disease (GD) is an inborn error of metabolism that affects the recycling of cellular glycolipids. It is an autosomal recessive inherited disorder of metabolism. The major clinical symptoms of GD include enlargement of the liver and spleen (hepatosplenomegaly), low number of red blood cells (anemia), easy bruising, and bone disease (bone pain and fractures). The diagnosis of GD is based on clinical symptoms and laboratory testing. A diagnosis of Gaucher disease is suspected in individuals who have bone problems, enlarged liver and spleen (hepatosplenomegaly), changes in red blood cell levels, easy bleeding and bruising from low platlets or signs of nervous system problems. Treatment is individualized for each patient depending on the type of Gaucher disease. Enzyme replacement therapy (ERT) has proven effective for individuals with Gaucher disease type 1.
Recent Developmental Activities in the Gaucher's Disease Treatment Landscape
• In April 2022, Sanofi initiated a parallel arm, Phase 3, double-blind,active-comparator, 2 arm study to evaluate the efficacy and safety of daily oral venglustat versus intravenous Cerezyme infusions every two weeks for improvement or stabilization of the neurological manifestations and maintenance of systemic disease stability in participants aged ≥12 and
• GPH301 is a next generation gene-edited autologous HSC product candidate leveraging the CCR5 locus technology for the treatment of Gaucher disease. With GPH301, a functional copy of the gene for glucocerebrosidase (GCase) is inserted into the chromosomal location of the CCR5 gene. The drug is in preclinical studies for the treatment of Gaucher's Disease.
• AVR-RD-02 is an ex vivo lentiviral gene therapy, designed using Avrobio's plato platform. The drug is in Phase I/II clinical studies for the treatment of Gaucher's Disease.
Gaucher's Disease Emerging Drugs Profile
• GPH301: Graphite Bio
• AVR-RD-02: AVROBIO
Gaucher's Disease Therapeutic Assessment
There are approx. 14+ key companies which are developing the therapies for Gaucher's Disease. The companies which have their Gaucher's Disease drug candidates in the most advanced stage, i.e. Phase I/II include, AVROBIO.
Get an overview of the Immune Thrombocytopenia Ongoing Clinical Trials Analysis @ https://www.delveinsight.com/report-store/gauchers-disease-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr
Scope of the Gaucher's disease Pipeline
• Coverage: Global
• Key Gaucher's disease Companies: Gain therapeutics, M6P Therapeutics, AvroBio, Orphazyme, Eli Lilly and Company, Sanofi, and others
• Key Gaucher's disease Therapies: GBA programme, M 011, AVR-RD-02, Arimoclomol, GBA1 Gene Therapy, Venglustat, and others
• Gaucher's disease Therapeutic Assessment: Gaucher's disease current marketed and Gaucher's disease emerging therapies
• Gaucher's disease Market Dynamics: Gaucher's disease market drivers and Gaucher's disease market barriers
Table of Content
1. Introduction
2. Executive Summary
3. Gaucher's Disease: Overview
4. Pipeline Therapeutics
5. Therapeutic Assessment
6. Gaucher's Disease - DelveInsight's Analytical Perspective
7. Late Stage Products (Phase III)
8. Eliglustat: Sanofi
9. Drug profiles in the detailed report…..
10. Mid Stage Products (Phase II)
11. Arimoclomol: Orphazyme
12. Drug profiles in the detailed report…..
13. Early Stage Products (Phase I/II)
14. AVR-RD-02: AVROBIO
15. Drug profiles in the detailed report…..
16. Preclinical and Discovery Stage Products
17. CAN103: CANbridge Life Sciences
18. Drug profiles in the detailed report…..
19. Inactive Products
20. Gaucher's Disease Key Companies
21. Gaucher's Disease Key Products
22. Gaucher's Disease- Unmet Needs
23. Gaucher's Disease- Market Drivers and Barriers
24. Gaucher's Disease- Future Perspectives and Conclusion
25. Gaucher's Disease Analyst Views
26. Appendix
Key Questions
Current Treatment Scenario and Emerging Therapies:
• How many companies are developing Gaucher's Disease drugs?
• How many Gaucher's Disease drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Gaucher's Disease?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Gaucher's 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 Gaucher's Disease and their status?
• What are the key designations that have been granted to the emerging drugs?
For further information on the Immune thrombocytopenia Pipeline therapeutics, reach out to Immune thrombocytopenia Treatment Landscape @ https://www.delveinsight.com/report-store/gauchers-disease-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr
Company Name: DelveInsight LLP
Contact Person: Yash Bhardwaj
Email: ybhardwaj@delveinsight.com
Phone: 09193216187
Address: 304 S. Jones Blvd #2432,
City: Las Vegas
State: United States
Country: India
Website: https://www.delveinsight.com/
About Us
DelveInsight is a Business Consulting and Market research company, providing expert business solutions for the healthcare domain and offering quintessential advisory services in the areas of R&D, Strategy Formulation, Operations, Competitive Intelligence, Competitive Landscaping, and Mergers & Acquisitions.
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