Gaucher’s Disease - Pipeline Insight, 2021

DelveInsights, Gauchers Disease - Pipeline Insight, 2021, report provides comprehensive insights about 14+ companies and 16+ pipeline drugs in Gauchers Disease 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

Gauchers Disease Understanding
Gauchers 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.

"Gauchers Disease - Pipeline Insight, 2021" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Gauchers Disease pipeline landscape is provided which includes the disease overview and Gauchers Disease treatment guidelines. The assessment part of the report embraces, in depth Gauchers Disease 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, Gauchers Disease 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 Gauchers Disease R&D. The therapies under development are focused on novel approaches to treat/improve Gauchers Disease.

Gauchers Disease Emerging Drugs Chapters
This segment of the Gauchers Disease report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, 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.

Gauchers Disease Emerging Drugs
GPH301: Graphite Bio
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 Gauchers Disease.

AVR-RD-02: AVROBIO
AVR-RD-02 is an ex vivo lentiviral gene therapy, designed using Avrobios plato platform. The drug is in Phase I/II clinical studies for the treatment of Gauchers Disease.
Further product details are provided in the report..

Gauchers Disease: Therapeutic Assessment
This segment of the report provides insights about the different Gauchers Disease drugs segregated based on following parameters that define the scope of the report, such as:
Major Players in Gauchers Disease
There are approx. 14+ key companies which are developing the therapies for Gauchers Disease. The companies which have their Gauchers Disease drug candidates in the most advanced stage, i.e. Phase I/II include, AVROBIO.

Phases
DelveInsights report covers around 16+ products under different phases of clinical development like
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
Gauchers Disease pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as
Oral
Parenteral
Intravitreal
Subretinal
Topical

Molecule Type
Products have been categorized under various Molecule types such as
Monoclonal Antibody
Peptides
Polymer
Small molecule
Gene therapy

Product Type
Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Gauchers Disease: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase III, II, I, preclinical and discovery stage. It also analyses Gauchers Disease 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 Gauchers Disease drugs.

Gauchers Disease Report Insights
Gauchers Disease Pipeline Analysis
Therapeutic Assessment
Unmet Needs
Impact of Drugs

Gauchers Disease Report Assessment
Pipeline Product Profiles
Therapeutic Assessment
Pipeline Assessment
Inactive drugs assessment
Unmet Needs

Key Questions
Current Treatment Scenario and Emerging Therapies:
How many companies are developing Gauchers Disease drugs?
How many Gauchers Disease drugs are developed by each company?
How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Gauchers Disease?
What are the key collaborations (IndustryIndustry, IndustryAcademia), Mergers and acquisitions, licensing activities related to the Gauchers 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 Gauchers Disease and their status?
What are the key designations that have been granted to the emerging drugs?

Key Players
CANbridge Life Sciences
AVROBIO
Gain Therapeutics
M6P Therapeutics
Denali Therapeutics
Graphite Bio
Sanofi
Freeline Therapeutics
Orphazyme
Sanofi
Prevail Therapeutics
Yuhan

Key Products
CAN103
AVR-RD-02
GT-02287
GT-02329
M-011
M-013
ETV:GCase
GPH 301
Venglustat
FLT 201
Arimoclomol
Eliglustat
PR-001
YHC-1116

Introduction
Executive Summary
Gauchers Disease: 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
Gauchers Disease DelveInsights Analytical Perspective
Late Stage Products (Phase III)
Comparative Analysis
Eliglustat: Sanofi
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report..
Mid Stage Products (Phase II)
Comparative Analysis
Arimoclomol: Orphazyme
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report..
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
CAN103: CANbridge Life Sciences
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report..
Inactive Products
Comparative Analysis
Gauchers Disease Key Companies
Gauchers Disease Key Products
Gauchers Disease- Unmet Needs
Gauchers Disease- Market Drivers and Barriers
Gauchers Disease- Future Perspectives and Conclusion
Gauchers Disease Analyst Views
Appendix

List Of Tables

Table 1 Total Products for Gauchers Disease
Table 2 Late Stage Products
Table 3 Mid Stage Products
Table 4 Early Stage Products
Table 5 Pre-clinical & Discovery Stage Products
Table 6 Assessment by Product Type
Table 7 Assessment by Stage and Product Type
Table 8 Assessment by Route of Administration
Table 9 Assessment by Stage and Route of Administration
Table 10 Assessment by Molecule Type
Table 11 Assessment by Stage and Molecule Type
Table 12 Inactive Products

List Of Figures

Figure 1 Total Products for Gauchers Disease
Figure 2 Late Stage Products
Figure 3 Mid Stage Products
Figure 4 Early Stage Products
Figure 5 Preclinical and Discovery Stage Products
Figure 6 Assessment by Product Type
Figure 7 Assessment by Stage and Product Type
Figure 8 Assessment by Route of Administration
Figure 9 Assessment by Stage and Route of Administration
Figure 10 Assessment by Molecule Type
Figure 11 Assessment by Stage and Molecule Type
Figure 12 Inactive Products

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