Gene Therapy for Mucopolysaccharidosis - A Pipeline Analysis Report

This pipeline analysis report provides detailed insights into the clinical trials landscape of the gene therapy for mucopolysaccharidosis including molecules at pre-clinical and various other development stages. The report also offers comprehensive information about the therapeutic assessment of the pipeline molecules based on various segmentations including route of administration.

Overview of the gene therapy pipeline for mucopolysaccharidosis

Mucopolysaccharidosis is a rare and autosomal recessive disease caused by an inherited deficiency of an enzyme that is involved in the degradation of acid glycosaminoglycans. Some of the symptoms of this disease include abnormalities of the skeleton, liver and spleen enlargement, heart abnormalities, and neurological complications. It has been observed that in the recent years, mucopolysaccharidoses I had the highest incidence and prevalence rate among the various types of mucopolysaccharidosis. Therapies already available for mucopolysaccharidosis are focused on slowing down its progression and currently, there is no permanent cure for this disease. The market research analysts have predicted that with the replacement gene therapy will emerge as one of the most promising approach to treat mucopolysaccharidoses and the global mucopolysaccharidoses market will witness significant growth in the coming years.

According to this pipeline analysis report, majority of molecules in the pipeline are targeted at mucopolysaccharidosis III A (sanfilippo type A syndrome) and five major companies are involved in the development of gene therapy molecules for this disease. The only company focusing on the development of treatment for mucopolysaccharidosis III B (sanfilippo type B syndrome) is Abeona Therapeutics.

Companies covered

This pipeline analysis report provides a detailed analysis of the companies that are actively involved in the development of gene therapy molecules for the treatment of mucopolysaccharidosis. In addition to providing detailed information on the various stages of molecules developed 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

• Sangamo Therapeutics


• Swedish Orphan Biovitrum


• uniQure

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

• Intravenous


• ICV


• Intracerebral


• Intracisternal

In the intravenous route of administration, the drug is directly delivered into the vein of the mucopolysaccharidosis gene therapy candidates. According to our pipeline analysis report, the maximum number of gene therapy molecules for the treatment of mucopolysaccharidosis are being developed as intravenous and ICV drugs.

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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