Final Report Summary - PONT (Disease therapeutic targets, multidisciplinary parallel validation, accelerated drug discovery)
Genomics approaches deliver hundreds of candidate new disease drug targets in a single experiment. However, these targets are poorly characterised, particularly with respect to the medical relevance, and current drug discovery processes are not optimised to handle the huge numbers of genome-based targets. PONT was designed to incorporate companies that have unique technological advantages at specific steps during the development of novel compounds.
Based on the integration of new technologies for aptamer-based compound screening combined with tissue-based target verification compounds for new (i.e. Rheb2, Ephrin-A3) as well as tricky targets (IGFR1, Farnesyltransferase) were evaluated for the treatment of hepatocellular carcinoma (HCC) and subsequently for other carcinomas. The selected aptamers for compound screening had been validated in cell culture models as well as in mouse models. Compounds selected from aptamer-based hit screening showed activity in cell culture as well as mouse models.
PONT could successfully prove that an aptamer-based approach for new or tricky drug targets can provide compounds with exciting and promising preclinical activity in reasonable timelines.
Based on the integration of new technologies for aptamer-based compound screening combined with tissue-based target verification compounds for new (i.e. Rheb2, Ephrin-A3) as well as tricky targets (IGFR1, Farnesyltransferase) were evaluated for the treatment of hepatocellular carcinoma (HCC) and subsequently for other carcinomas. The selected aptamers for compound screening had been validated in cell culture models as well as in mouse models. Compounds selected from aptamer-based hit screening showed activity in cell culture as well as mouse models.
PONT could successfully prove that an aptamer-based approach for new or tricky drug targets can provide compounds with exciting and promising preclinical activity in reasonable timelines.