By Cormac Sheridan
BioWorld International Correspondent
Belgian functional genomics firm Galapagos Genomics NV scored two target discovery deals based on its recently completed SilenceSelect platform, which uses small interfering RNAs to identify genes involved in disease-related processes.
Michelen-based Galapagos disclosed a deal with Boehringer Ingelheim, of Ingelheim, Germany, on Tuesday. The second deal will be disclosed Thursday and is with a U.S. pharmaceutical company, the company said. Further information on that deal was not disclosed, but is said to be significant in size.
Specific financial terms of the Boehringer deal also were not disclosed, but Galapagos Genomics will receive an up-front technology access fee, plus additional license and option fees over the life of the agreement. It is focused on a particular viral pathogen, the identity of which the partners are not disclosing.
"They have an assay system where they have engineered a cell with the replication system of the virus," Galapagos CEO Onno van de Stolpe told BioWorld International.
Galapagos Genomics will use the assay system to screen for proteins involved in the replication process. Its SilenceSelect platform consists of a collection of modified adenoviral vectors that encode hairpin RNA structures, which are then processed to give rise to functional small interfering RNA (siRNA) molecules once they are expressed inside the cell. These down-regulate the expression of more than 4,000 human genes encoding targets such as kinases, phosphatases, G protein-coupled receptors, transporters, proteases, receptors, nuclear hormone receptors, ion channels and phosphodiesterases - what van de Stolpe refers to as the "druggable human genome."
The company's viral platform offers advantages in terms of efficiency and consistency over reagent-based methods for introducing siRNAs, van de Stolpe said, as RNA transfection rates vary across different cell types. The duration of the effect also is longer, as the viral vector persists for up to one week, continuing to express siRNA during that time. In contrast, individual siRNA molecules are metabolized within eight hours once inside the cell, he said.
Van de Stolpe said pharmaceutical firms are taking a renewed interest in knockdown technologies because they directly mimic the action of small molecules on drug targets.
"For target discovery and validation, siRNA is much more reliable than antisense - or ribozymes for that matter," he said.
The agreements are the company's first to involve the complete platform, although earlier this year it did carry out a validation assignment for another pharmaceutical company that involved a limited subset of genes.
"I think the first deals we're making with the platform will show the community we have something valuable for target discovery here," van de Stolpe said.
The company has raised €32 million since it was established in 1999. It closed a €23 million round last year. "We still have about two-and-a-half years in cash left," van de Stolpe said. The company plans to seek further funding toward the end of 2004.