Advancing the Research and Development of Enzyme Replacement Therapies for Lysosomal Storage Diseases.
enzyme replacement therapy
lysosomal storage diseases
lysosomes
pipeline
rare diseases
Journal
GEN biotechnology
ISSN: 2768-1556
Titre abrégé: GEN Biotechnol
Pays: United States
ID NLM: 9918400388506676
Informations de publication
Date de publication:
Apr 2022
Apr 2022
Historique:
entrez:
16
6
2022
pubmed:
17
6
2022
medline:
17
6
2022
Statut:
ppublish
Résumé
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 we need to develop a pipeline of proteins analogous to what has been created in some open science efforts as well as apply technologies to decrease manufacturing costs. In this Perspective, we illustrate the opportunity to fill the rare LSD treatment gap with ERTs while gene therapies are in development for these life-shortening diseases.
Identifiants
pubmed: 35706761
doi: 10.1089/genbio.2021.0013
pmc: PMC9192161
mid: NIHMS1804591
doi:
Types de publication
Journal Article
Langues
eng
Pagination
156-162Subventions
Organisme : NINDS NIH HHS
ID : R43 NS107079
Pays : United States
Organisme : NINDS NIH HHS
ID : R44 NS107079
Pays : United States
Déclaration de conflit d'intérêts
Conflicts of interest S.E. is owner, and A.P are employees of Collaborations Pharmaceuticals, Inc.
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