
Gaucher’s disease type I - Pipeline Insight, 2024
Description
Gaucher’s disease type I - Pipeline Insight, 2024
DelveInsight’s, “Gaucher’s disease type I - Pipeline Insight, 2024,” report provides comprehensive insights about 4+ companies and 4+ pipeline drugs in Gaucher’s disease type I 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 I: Overview
Gaucher disease (GD) type 1 is the most prevalent form of the disease in western countries, making up about 95 percent of cases there. GD type 1 is a lysosomal storage disease caused by a mutation in the GBA gene (localized to 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, of the spleen and the bone marrow (Gaucher cells). Diagnostic methods involve ultrasound and magnetic resonance imaging (MRI) for the initial evaluation and subsequent monitoring of hepatosplenomegaly, radiography and bone scintigraphy to detect bone lesions and complications, osteodensitometry for the evaluation of osteopenia of the lumbar spine and femoral neck, and cardiac ultrasound for the detection of pulmonary arterial hypertension. The standard treatment for GD type 1 is enzyme substitution therapy, administered intravenously (e.g.: imiglucerase with European marketing authorization (MA) since 1997 and velaglucerase with a MA since 2010). Substrate reduction therapy (miglustat), administered orally, provides an alternative second-line treatment when enzyme substitution therapy is not suitable. Bisphophonates can also be proposed to prevent bone complications.
""Gaucher’s disease type I - 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 I pipeline landscape is provided which includes the disease overview and Gaucher’s disease type I treatment guidelines. The assessment part of the report embraces, in depth Gaucher’s disease type I 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 I 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 I R&D. The therapies under development are focused on novel approaches to treat/improve Gaucher’s disease type I.
This segment of the Gaucher’s disease type I 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 I Emerging Drugs
- GPH301: Graphite Bio
- CAN103: CANbridge Pharmaceuticals
- AVR-RD-02: AVROBIO
Further product details are provided in the report……..
Gaucher’s disease type I: Therapeutic Assessment
This segment of the report provides insights about the different Gaucher’s disease type I drugs segregated based on following parameters that define the scope of the report, such as:
- Major Players in Gaucher’s disease type I
- 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 I: 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 I 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 I drugs.
Gaucher’s disease type I Report Insights
- Gaucher’s disease type I 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 I drugs?
- How many Gaucher’s disease type I 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 I?
- What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Gaucher’s disease type I 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 I and their status?
- What are the key designations that have been granted to the emerging drugs?
- Graphite Bio
- CANbridge Pharmaceuticals
- Freeline Therapeutics
- ILIAS Biologics
- AVROBIO, Inc.
- GPH301
- CAN103
- FLT 201
- Exosome-based Gaucher's disease Type-I and Gaucher's disease Type-III enzyme therapeutic
- AVR-RD-02
Table of Contents
40 Pages
- Introduction
- Executive Summary
- Gaucher’s disease type I: 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 I – DelveInsight’s Analytical Perspective
- Early Stage Products (Phase I/II)
- Comparative Analysis
- AVR-RD-02: AVROBIO
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Preclinical and Discovery Stage Products
- Comparative Analysis
- GPH301: Graphite Bio
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Inactive Products
- Comparative Analysis
- Gaucher’s disease type I Key Companies
- Gaucher’s disease type I Key Products
- Gaucher’s disease type I- Unmet Needs
- Gaucher’s disease type I- Market Drivers and Barriers
- Gaucher’s disease type I- Future Perspectives and Conclusion
- Gaucher’s disease type I Analyst Views
- Gaucher’s disease type I Key Companies
- Appendix
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