Gaucher’s Disease - Drug Pipeline Landscape, 2023

Gaucher's disease, or Gaucher disease (GD), is an inherited genetic disorder of the lysosomal storage that is caused by a genetic defect of the lysosomal enzyme "glucocerebrosidase." This enzyme accumulates in cells and certain organs, including the spleen and liver. The condition is mainly of 3 types: Gaucher disease type 1, Gaucher disease type 2, and Gaucher disease type 3.

Gaucher Disease's main cause is mutations in the GBA1 gene, resulting in a lysosomal deficiency.

Gaucher Disease symptoms include enlargement of the liver and spleen, a low number of red blood cells, easy bruising, and bone disease (bone pain and fractures).

Gaucher Disease is diagnosed by clinical symptoms and laboratory tests like an enzyme test called a beta-glucosidase leukocyte (BGL) test.

Gaucher Disease is treated with enzyme replacement therapy, a bone marrow transplant and procedures like spleen removal.

Report Highlights

Global Insight Service’s, Gaucher’s Disease - Drug Pipeline Landscape, 2023 report provides an overview of the Gaucher’s Disease pipeline drugs. This report covers detailed insights on Gaucher’s Disease drugs under development, assessment by target, mechanism of action, route of administration and molecule type.Product pipeline by companies, stage of development and key regulatory designations, deals and milestones have been presented to provide insights and thus help industry participants in their decision making. Gaucher’s Disease pipeline report helps gain insights on drugs which are under development stage of drug development process across globally.

Methodology

The research process includes extensive secondary research on public domain and other authentic sources to add or update the pipeline products information. The secondary research sources include, but are not limited to company websites, annual reports, financial reports, company pipeline chart, investor presentations and SEC filings, journals and conferences, and clinical trials registries.

Scope

The pipeline landscape report provides analysis of pipeline products based on several stages of development ranging from Discovery to Pre-Registration. The report provides a review of pipeline therapeutics by companies based on information derived from company and industry-specific sources. The pipeline report covers assessment of therapeutics by mechanism of action (MoA), drug target, route of administration (RoA) and molecule type. Comprehensive profiles of the pipeline products with details such as company overview, development stage; molecule type, target, mechanism of action, route of administration, dosage form, regulatory designations, key deals, clincial trials, and key upcoming milestones are included.

Reasons to Buy

Helps to find and recognize significant therapeutics under development. Thorough understanding of pipeline structure and helps in developing corrective measures for pipeline projects.

Effective R&D strategies can be developed with deep knowledge of competitor information, analysis, and insights.

Plan collaborations with various industry partners that have role in some or the other stage of drug development such as contract manufacturing, co-development, contract research organization and commercialization etc.

Helps to create in-licensing and out-licensing opportunities by identifying prospective partners with attractive projects to expand business potential and scope.

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