The major objective of this product group is to provide access to an integrated infrastructure that can aid the development of identified lead compounds, from optimization of the design and formulation, product synthesis, in vivo pre-clinical testing up to the initiation of clinical testing. 

Typically optimization of a lead compound requires successive steps of chemical synthesis which are heavily influenced not only by the results of the original screening procedures, but also by the data obtained after more complex and intermediate assays. In today's academic research environment, various structural and infrastructural shortcomings severely compromise and/or retard the translation of scientific findings into clinical applications. Other deficits are clearly linked to the specificity of the development chain.

In order to be able to provide integrated transantional services, the group has harmonised compound screening procedures, improved the quality and quantity of the available technologies across all EATRIS centres for in vitro, in vivo and toxicity testing. We make use in first instance of profiling assays that can be used for high throughput screening, based on e.g. automated image analysis of phenotypic effects, and in silico algorithms to predict effectiveness of the compounds. For all assays SOPs are written, with several pilot studies to assess the quality and quantity of these assays. The outcomes are used to assess the service to take a candidate compound into the next phase of optimizing the compound itself and the synthesis under the required regulations.

The group incorporates the possibility to develop a biomarker to guide application of targeted therapies in close collaboration with the Product Group Biomarkers. With Product Group Imaging/Tracers there is two-way collaboration. For the in vitro and in vivo models we use  a pipeline of developing compounds with an imaging linker , including imaging biomarkers (based on tracer compounds) for the use in early clinical development phases to: a) confirm drug distribution (i.e. brain penetration); b) optimize posology; c) confirm early proof of concept; d) help identify patients who are most likely to benefit, and e) make the transition to phase III clinical trials more valid, rapid and improve the success rate.

In parallel there is a platform for in vivo testing. The available animal models will be cataloged and a SOP for in vivo and toxicity testing in the different types of animal models will be written and tested. The chosen animal models include rodents for in vivo testing to rodents, dogs etc for toxicity testing. To run these pilots we select a number promising lead compounds in collaboration with other initiatives.

This group also focuses on innovative therapeutic approaches such as the use of bioactive peptides and nanoparticles for brain drug targeting. Peptides are short aminoacid polymers with a defined primary structure and can be easily synthesized with FMOC chemistry. This type of compounds has enormous potential, particularly within the frame of targeted therapy.  Moreover, a growing number of engineered nanoparticles (NP) are now being used in theranostic applications. This avenue will also form an area of interest.