Advancing the Research and Development of Enzyme Replacement Therapies for Lysosomal Storage Diseases.

ABSTRACT: With the increasing interest in developing gene therapies for rare diseases, it is easy to overlook that there are numerous rare lysosomal storage diseases (LSD) with treatments that have been approved by regulatory agencies in the United States and Europe. These primarily consist of enzyme replacement therapies (ERT), which are recombinant human proteins that are delivered for the life of the patient via different routes and may have distinct safety and distribution advantages over gene therapies. The research and development of ERT is a lengthy and expensive process, which is usually performed in academic laboratories before transfer to pharmaceutical companies and is hence a process ripe for disruption. There may still be considerable scientific and investment potential for ERT, however

SUBMITTER: Puhl AC 

PROVIDER: S-EPMC9192161 | biostudies-literature | 2022 Apr

REPOSITORIES: biostudies-literature