Mucopolysaccharidosis - Drug Pipeline Landscape, 2023

Mucopolysaccharidosis is a group of metabolic inherited lysosomal storage disorder caused by a deficiency of alpha-L-iduronidase, a lysosomal enzyme normally required for the breakdown of certain complex carbohydrates known as glycosaminoglycans (GAGs). There are seven types of mucopolysaccharidosis : mps type I (hurler syndrome), mps type II (hunter syndrome), mps type III (sanfilippo syndrome), mps type IV (morquio syndrome), mps type VI (maroteaux-lamy syndrome), mps type VII (sly syndrome), and mps type IX (hyaluronidase deficiency).

Mucopolysaccharidosis is caused by the absence or malfunctioning of certain enzymes in the body needs to break down molecules called glycosaminoglycans.

Mucopolysaccharidosis main symptoms are abnormal bones in the spine, claw hands, cloudy corneas, deafness, heart valve problems, liver and spleen enlargement, and changing facial features.

Mucopolysaccharidosis is diagnosed by low iduronate 2-sulfatase enzyme activity and elevated plasma and or urine GAGs.

Mucopolysaccharidosis is treated by enzyme replacement therapy (ERT) and/or hematopoietic stem cell transplantation.

Report Highlights

Global Insight Service’s, Mucopolysaccharidosis - Drug Pipeline Landscape, 2023 report provides an overview of the Mucopolysaccharidosis pipeline drugs. This report covers detailed insights on Mucopolysaccharidosis 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. Mucopolysaccharidosis 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, clinical 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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