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Lysosomal Storage Disorder Therapeutics Pipeline has Maximum Drugs Being Developed as Gene Therapy

Published Date:   June 2017

According to a new research report “Lysosomal Storage Disorder Therapeutics Pipeline Analysis, 2017 - Clinical Trials & Results, Patent, Designation, Collaboration, and Other Developments” published by P&S Intelligence, Lysosomal Storage Disorder currently exhibits a strong pipeline with 74 drug candidates.

Browse Report Description at:

Lysosomal Storage Disorder therapeutics pipeline in 2017

The study analyzed that the lysosomal storage disorder therapeutics pipeline comprises approximately 74 drug candidates in different stages of development.

Insights on pipeline segments

According to the research findings, most of the drug candidates for lysosomal storage disorder pipeline are being developed to be administered by intravenous route.

CHART technology platform for lysosomal storage disorder drug development

Amicus Therapeutics, Inc. is using chaperone advanced replacement therapy (CHART) technology platform for the development of their drug candidates for the treatment of Fabry disease, a type of lysosomal storage disorder. In a chaperone-advanced replacement therapy, a unique pharmacological chaperone, binds to the infused therapeutic enzyme, stabilizing the enzyme in its properly folded and active form. This proposed CHART mechanism may allow for enhanced tissue uptake, greater lysosomal activity, more reduction of storage material, and lower immunogenicity compared to ERT alone.

Natural Sources used for the development of drugs in the Lysosomal Storage Disorder Therapeutics Pipeline

The research also found that various companies use natural sources for the development of lysosomal storage disorder therapeutics pipeline. Protalix BioTherapeutics, Inc. is developing their drug candidates, obtained from a natural source, for the treatment of Fabry disease.

Some of the other key players developing drugs for the treatment of lysosome storage disorder include GlaxoSmithKline plc, Sanofi Genzyme, Amicus Therapeutics, Inc. and others.

Lysosomal Storage Disorder Therapeutics Pipeline Analysis

  • By Phase
  • By Route of Administration
  • By Molecule Type
  • By Company