The "Gene
Therapy for Mucopolysaccharidosis - A Pipeline Analysis Report"
report has been added to ResearchAndMarkets.com's
offering.
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.
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.
For more information about this report visit https://www.researchandmarkets.com/research/7h9rvb/gene_therapy_for?w=4
View source version on businesswire.com: https://www.businesswire.com/news/home/20180615005301/en/
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Related
Topics: Genomics,
Immune
Disorders Drugs