Gene Therapy for Mucopolysaccharidosis - A Pipeline Analysis Report

Overview of the gene therapy pipeline for mucopolysaccharidosis

It has been observed that mucopolysaccharidosis (include a group of more than 40 genetic disorders) are part of the lysosomal storage diseases, which are caused by an inherited deficiency of an enzyme involved in the degradation of acid glycosaminoglycans (GAGs), previously known as mucopolysaccharides. All mucopolysaccharidoses are rare, autosomal recessive, except for mucopolysaccharidosis II, which are X-linked. The common symptoms of mucopolysaccharidosis are multiple organ involvements, rough facial features, and abnormalities of the skeleton, especially joint problems. In addition, symptoms of mucopolysaccharidosis also include short stature, heart abnormalities, breathing irregularities, liver and spleen enlargement, and/or neurological complications. Replacement gene therapy is emerging as the most efficient approach for the treatment of mucopolysaccharidosis.

According to this pipeline analysis report, most of the gene therapy molecules in the pipeline are being developed for mucopolysaccharidosis III A. Our market research analysts have also identified that most of these molecules are in the pre-clinical development stage and a considerable number of molecules have been discontinued from development.

Companies covered

This pipeline analysis report provides a detailed analysis of the companies that are involved in the development of gene therapy molecules for the treatment of mucopolysaccharidosis. In addition to providing information on the various stages of molecules developed by companies for different indications, this pipeline analysis report also provides information about the gene therapy molecules discontinued by companies.

Some of the companies covered in this pipeline analysis report are 

• Abeona Therapeutics
• ESTEVE
• Immusoft

Therapeutic assessment of the gene therapy pipeline for mucopolysaccharidosis by route of administration

• Intravenous
• ICV
• Intracerebral
• Intracisternal
• Unspecified (parenteral)

The intravenous route of administration (ROA) involves the application of the drug directly into the vein and in the ICV technique, the molecules are directly administered into the CSF in cerebral ventricles, which will have a more direct effect on the target cells.

Therapeutic assessment of the gene therapy pipeline for mucopolysaccharidosis by therapy

• Replacement gene therapy

According to this pipeline analysis report, all the molecules that are currently in the gene therapy pipeline for mucopolysaccharidosis are being developed as replacement gene therapy drugs and most of these molecules are in the pre-clinical stage of development.

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Key questions answered in the report include

• What are the gene therapy molecules in the various development stages for mucopolysaccharidosis?
• What are the companies that are currently involved in the development of gene therapy molecules for mucopolysaccharidosis?
• Insight into discontinued/inactive molecules with appropriate reasoning?
• What are the major regulatory authorities approving drugs in various regions?
• Detailed profiling of each active molecule

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