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. Technavio's 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.
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 -
Swedish Orphan Biovitrum
Therapeutic assessment of the gene therapy pipeline for mucopolysaccharidosis by route of administration
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.
Secondary Research Information is collected from a number of publicly available as well as paid databases. Public sources involve publications by different associations and governments, annual reports and statements of companies, white papers and research publications by recognized industry experts and renowned academia etc. Paid data sources include third party authentic industry databases.
Once data collection is done through secondary research, primary interviews are conducted with different stakeholders across the value chain like manufacturers, distributors, ingredient/input suppliers, end customers and other key opinion leaders of the industry. Primary research is used both to validate the data points obtained from secondary research and to fill in the data gaps after secondary research.
The market engineering phase involves analyzing the data collected, market breakdown and forecasting. Macroeconomic indicators and bottom-up and top-down approaches are used to arrive at a complete set of data points that give way to valuable qualitative and quantitative insights. Each data point is verified by the process of data triangulation to validate the numbers and arrive at close estimates.
The market engineered data is verified and validated by a number of experts, both in-house and external.
REPORT WRITING/ PRESENTATION
After the data is curated by the mentioned highly sophisticated process, the analysts begin to write the report. Garnering insights from data and forecasts, insights are drawn to visualize the entire ecosystem in a single report.
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