
Gaucher's Disease Type III - Pipeline Insight, 2024
Description
Gaucher's Disease Type III - Pipeline Insight, 2024
DelveInsight’s, “Gaucher’s disease type III - Pipeline Insight, 2024,” report provides comprehensive insights about 7+ companies and 7+ pipeline drugs in Gaucher’s disease type III 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.
Geography Covered
- Global coverage
Gaucher’s disease type III: Overview
Gaucher disease type 3 is the subacute neurological form of Gaucher disease characterized by progressive encephalopathy and associated with the systemic manifestations (organomegaly, bone involvement, cytopenia) of GD type 1. The annual incidence of GD is about 1/60,000 and the prevalence is approximately 1/100,000. GD type 3 accounts for 5% of all patients with GD. GD type 3 is a lysosomal storage disease caused by a mutation in the GBA gene (1q21) that codes for the lysosomal enzyme, glucocerebrosidase. The deficiency in glucocerebrosidase leads to the accumulation of glucosylceramidase (or beta-glucocerebrosidase) deposits in the cells of the reticuloendothelial system of the liver, spleen and the bone marrow (Gaucher cells). The treatment for patients with GD type 3 exhibiting clinically significant non neurological manifestations is enzyme substitution therapy (imiglucerase with marketing authorization (MA) since 1997). It appears to slow progression of the neurological symptoms and is effective against the systemic manifestations.
""Gaucher’s disease type III - Pipeline Insight, 2024"" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Gaucher’s disease type III pipeline landscape is provided which includes the disease overview and Gaucher’s disease type III treatment guidelines. The assessment part of the report embraces, in depth Gaucher’s disease type III commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Gaucher’s disease type III collaborations, licensing, mergers and acquisition, funding, designations and other product related details.
Report Highlights
- The companies and academics are working to assess challenges and seek opportunities that could influence Gaucher’s disease type III R&D. The therapies under development are focused on novel approaches to treat/improve Gaucher’s disease type III.
This segment of the Gaucher’s disease type III report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.
Gaucher’s disease type III Emerging Drugs
- Venglustat: Sanofi Genzyme
- CAN103: CANbridge Pharmaceuticals
Further product details are provided in the report……..
Gaucher’s disease type III: Therapeutic Assessment
This segment of the report provides insights about the different Gaucher’s disease type III drugs segregated based on following parameters that define the scope of the report, such as:
- Major Players in Gaucher’s disease type III
- Phases
- 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
- Oral
- Parenteral
- intravenous
- Subcutaneous
- Topical.
- Molecule Type
- Monoclonal Antibody
- Peptides
- Polymer
- Small molecule
- Gene therapy
- Product Type
Gaucher’s disease type III: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Gaucher’s disease type III therapeutic drugs key players involved in developing key drugs.
Pipeline Development Activities
The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Gaucher’s disease type III drugs.
Gaucher’s disease type III Report Insights
- Gaucher’s disease type III Pipeline Analysis
- Therapeutic Assessment
- Unmet Needs
- Impact of Drugs
- Pipeline Product Profiles
- Therapeutic Assessment
- Pipeline Assessment
- Inactive drugs assessment
- Unmet Needs
Current Treatment Scenario and Emerging Therapies:
- How many companies are developing Gaucher’s disease type III drugs?
- How many Gaucher’s disease type III 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 type III?
- What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Gaucher’s disease type III 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 type III and their status?
- What are the key designations that have been granted to the emerging drugs?
- Sanofi Genzyme
- CANbridge Pharmaceuticals
- AVROBIO
- Orphazyme
- KemPharm
- Graphite Bio
- Venglustat
- CAN103
- AVR RD 06
- Eliglustat
- Arimoclomol
- GPH 301
Table of Contents
60 Pages
- Introduction
- Executive Summary
- Gaucher’s disease type III: Overview
- Causes
- Mechanism of Action
- Signs and Symptoms
- Diagnosis
- Disease Management
- Pipeline Therapeutics
- Comparative Analysis
- Therapeutic Assessment
- Assessment by Product Type
- Assessment by Stage and Product Type
- Assessment by Route of Administration
- Assessment by Stage and Route of Administration
- Assessment by Molecule Type
- Assessment by Stage and Molecule Type
- Gaucher’s disease type III – DelveInsight’s Analytical Perspective
- Late Stage Products (Phase III)
- Comparative Analysis
- Venglustat: Sanofi Genzyme
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Mid Stage Products (Phase I/II)
- Comparative Analysis
- CAN103: CANbridge Pharmaceuticals
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Preclinical and Discovery Stage Products
- Comparative Analysis
- AVR RD 06: AVROBIO
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Inactive Products
- Comparative Analysis
- Gaucher’s disease type III Key Companies
- Gaucher’s disease type III Key Products
- Gaucher’s disease type III- Unmet Needs
- Gaucher’s disease type III- Market Drivers and Barriers
- Gaucher’s disease type III- Future Perspectives and Conclusion
- Gaucher’s disease type III Analyst Views
- Gaucher’s disease type III Key Companies
- Appendix
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