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Project

Qualification of the juvenile minipig for pediatric safety testing of single stranded oligonucleotides (SSOs).

Single stranded oligonucleotides (SSOs) are a new class of drugs that are used to inhibit the expression of specific proteins by hybridizing to specific mRNAs, e.g. in cancer cells. For adult indications, non-human primates (NHPs) are often considered to be the non-rodent species of choice for the safety assessment of SSOs, but recently the Göttingen minipig has been proven to be a good alternative. This animal model also has several ethical and practical advantages over NHPs for pediatric safety testing of SSOs, but the juvenile Göttingen minipig has not yet been characterized for this purpose. In the adult population, SSOs distribute from the circulation into tissues within minutes to a few hours after systemic administration. The aim of this project is to investigate these processes in juvenile Göttingen minipigs. These data will be very useful for the interpretation of pediatric safety data with new SSOs.
Date:15 Jul 2020 →  14 Jul 2021
Keywords:MODEL SYSTEMS, OLIGONUCLEOTIDES, PEDIATRICS
Disciplines:Toxicology and toxinology not elsewhere classified, Paediatrics