Gaucher’s Disease pipeline analysis shows tremendous growth opportunities for the upcoming years in the domain
DelveInsight’s Gaucher’s Disease pipeline report provides comprehensive insights into the current clinical development scenario and growth prospects in the Gaucher’s disease market. A detailed picture of Gaucher’s disease pipeline landscape is provided, including a disease overview and treatment guidelines for Gaucher’s disease.
Some of the Key Highlights from the Gaucher’s Disease Pipeline Report
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The Gaucher’s Disease pipeline comprises therapies that are in different stages of the clinical phase include Venglustat, Arimoclomol, PR-001, AVR-RD-02, M 011, GBA programme, and others that are expected to be available for Gaucher’s Disease treatment soon.
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Several pharmaceutical companies are evaluating their drug candidates for Gaucher’s Disease treatment include Sanofi, Orphazyme, Prevail Therapeutics, AvroBio, M6P Therapeutics, Gain therapeutics, among others.
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The therapies that are in late phase (Phase II/III) of development for Gaucher’s Disease include Venglustat (Sanofi), and others whereas Arimoclomol (Orphazyme) is in Phase II of Gaucher’s Disease clinical trials.
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Some therapies such as M 011, GBA programme, along with others are still in the early phase of development i.e. preclinical stage for Gaucher’s Disease treatment.
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Gaucher’s Disease: Overview
Gaucher Disease is a rare, inherited metabolic disorder characterized by a lack of the enzyme glucocerebrosidase, which causes an accumulation of harmful amounts of certain fats (lipids), specifically the glycolipid glucocerebroside, throughout the body, particularly in the bone marrow, spleen, and liver. Gaucher disease symptoms and physical findings vary greatly from patient to patient. There are three types of Gaucher’s Disease- Gaucher Disease Type 1, Gaucher Disease Type 2, and Gaucher Disease Type 3.
Gaucher’s Disease Symptoms
Gaucher’s Disease symptoms differ from person to person. Many people’s symptoms start in childhood. Some people experience only minor symptoms. Gaucher’s Disease symptoms can include:
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Enlarged Spleen
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Enlarged Liver
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Eye Movement Disorders
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Yellow Spots in the Eyes
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Seizures
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Bone Pain and Fracture
Gaucher’s Disease Treatment
Gaucher Disease Type 1 is treatable with regular therapy. Gaucher Disease treatments either increase enzyme levels or decrease the fatty substance that accumulates in the body as a result of Gaucher Disease. The neurological damage caused by Gaucher Disease Types 2 and 3 has no treatment. Gaucher’s Disease treatments available include enzyme replacement therapy (ERT) and substrate reduction therapy (SRT).
Gaucher’s Disease Pipeline Analysis: Drug Profiles
Venglustat: Sanofi
Product Description
Venglustat developed by Sanofi is a small-molecule glucosylceramide synthase (GCS) inhibitor that is designed to reduce the production of glucosylceramide (GL-1) and thus the formation of glucosylceramide-based glycosphingolipids.
Phase II/III
NCT02843035: Sanofi launched a three-part study in January 2017 to assess the efficacy and safety of Venglustat in combination with Cerezyme in adult and pediatric patients with Gaucher disease type 3 (GD3) receiving open-label long-term treatment.
Gaucher’s Disease Pipeline Therapies and Key Companies
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Venglustat: Sanofi
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Arimoclomol: Orphazyme
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PR-001: Prevail Therapeutics
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AVR-RD-02: AvroBio
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M 011: M6P Therapeutics
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GBA programme: Gain therapeutics
Get more details on the emerging therapies @ Gaucher’s Disease Treatment Gene Therapy
Gaucher’s Disease Therapeutics Assessment
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By Product Type
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Monotherapy
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Combination Therapy
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By Stage
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Discovery
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Pre-Clinical
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Phase I
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Phase II
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Phase III
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Pre-registration
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By Route of Administration
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Inhalation
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Intravenous
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Oral
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Subcutaneous
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By Molecule Type
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Small Molecule
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Stem Cell Therapy
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Gene Therapy
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Targets
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Immune System
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Multiple Kinase
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By Mechanism of Action
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Protease Inhibitors
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Immunomodulatory
Scope of the Report
Coverage: Global
Gaucher’s Disease Key Players: Sanofi, Orphazyme, Prevail Therapeutics, AvroBio, M6P Therapeutics, Gain therapeutics, among others
Gaucher’s Disease Pipeline Therapies: Venglustat, Arimoclomol, PR-001, AVR-RD-02, M 011, GBA programme, and others
Table of Contents
1. |
Introduction |
2. |
Executive Summary |
3. |
Gaucher’s Disease: Overview |
4. |
Gaucher’s Disease Pipeline Therapeutics |
5. |
Gaucher’s Disease Late Stage Products (Phase III) |
6. |
Gaucher’s Disease Mid Stage Products (Phase II/III) |
7. |
Gaucher’s Disease Mid Stage Products (Phase II) |
8. |
Gaucher’s Disease Early Stage Products (Phase I/II) |
9. |
Gaucher’s Disease Early stage products (Phase I/II) |
10. |
Gaucher’s Disease Preclinical and Discover Stage Products |
11. |
Gaucher’s Disease Preclinical and Discover Stage Products |
12. |
Gaucher’s Disease Therapeutic Assessment |
13. |
Gaucher’s Disease Inactive Products |
14. |
Gaucher’s Disease Company-University Collaborations (Licensing/Partnering) Analysis |
15. |
Gaucher’s Disease Key Companies |
16. |
Gaucher’s Disease Key Products |
17. |
Gaucher’s Disease- Unmet Needs |
18. |
Gaucher’s Disease- Market Drivers and Barriers |
19. |
Gaucher’s Disease- Future Perspectives and Conclusion |
20. |
Gaucher’s Disease Analyst Views |
21. |
Appendix |
22. |
About DelveInsight |
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Key questions answered in the Gaucher’s Disease Pipeline Report
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What are the treatment options for Gaucher’s Disease?
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How many pharmaceutical companies are working on Gaucher’s Disease treatments?
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Which of these companies’ pharmaceuticals is the most commonly used?
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What is the total number of Gaucher’s Disease medications produced by each company?
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How many drugs in the early, mid, or late stages of development for Gaucher’s Disease are there?
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How many of the current therapies in development can be used alone or in combination with other treatments?
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What are the most significant industry-industry and industry-academy collaborations, mergers and acquisitions, and licensing practices that have an impact on Gaucher’s Disease?
Related Reports
Gaucher Disease Type 1 Pipeline Insights
Get a comprehensive analysis of Gaucher Disease Type 1 pipeline therapies and key companies including Sanofi, Orphazyme, Prevail Therapeutics, among others.
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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