This task aims to directly or indirectly target intracellular processes deregulated by mutant proteins or as yet unknown factors. Partners will use and further improve already established drug screening platforms to identify compounds and drugs capable of inducing differentiation in different types of CNP.

This task will apply de novo protein design to create granulopoietic proteins and will test them for therapeutic efficacy in various CNP using primary hematopoietic stem cells, animal models and organoids.

This task will specifically focus on (a) the detailed design of several clinical trials including the description of the investigational medicinal product and the study protocol, the eligibility criteria for patient selection, the adverse event monitoring/reporting, the management of data according to the GDPR and national regulations, the informed consents for children and adults; (b) the preparation of regulatory and ethical requirements for the EMA approval of orphan drug usage and/or application of newly developed therapies including gene therapy; (c) the identification of centres for the implementation of multi-center, international clinical trials.