Research team
Expertise
Qualification of the Juvenile Göttingen Minipig for Pediatric Safety Testing of Single Stranded Oligonucleotides (SSOs) Single stranded oligonucleotide (SSO) therapeutics is a promising drug modality that enables selective modulation of currently difficult or even undruggable targets (e.g. cancer). Recently, the adult minipig has been characterized to be a suitable alternative non-rodent model in the adult safety assessment of SSOs as it has multiple ethical, and practical advantages over NHPs and dogs due to its favorable biological characteristics. Therefore, extending the work to qualify the juvenile minipig for pediatric safety testing of SSOs has been suggested. The aim of which is to assess whether differences in expression and activity of several nucleases, albumin concentrations, platelet factors, and blood-brain barrier occur in juvenile Göttingen Minipigs compared to adults and whether these can explain potential differences in in vivo toxicity to SSOs.
Qualification of the juvenile minipig for pediatric safety testing of single stranded oligonucleotides (SSOs).
Abstract
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.Researcher(s)
- Promoter: Van Cruchten Steven
- Fellow: Valenzuela Allan
Research team(s)
Project type(s)
- Research Project