The partners at the Hamburg site have an internationally visible track record in interdisciplinary pediatric research with a special focus on rare diseases, covering the entire value chain of therapeutic innovation, including genetic protein folding disorders, lysosomal diseases, immunology, gene therapy and genome editing technologies. Systems medicine in Hamburg is a founding member of one of the first centers for applied biomedical AI in Germany and is deeply embedded in clinical research and routine. The Hamburg site also has a development platform for small molecules. The platform has its own chemical library, which includes FDA/EMA-approved drugs, natural compounds and new chemical entities. The platform is operated by a cross-functional team with expertise in translational medicine, biochemistry, medicinal chemistry, pharmacology, bioinformatics and project management. The platform coupled with the team’s operational experience will enable rapid and successful integration of drug development campaigns from the various research areas and work programs of the DZKJ. The DZKJ infrastructure is additionally supported by a well-equipped research facility within the UKE research campus (University Children’s Research at Kinder-UKE, UCR), the newly established Pediatric University Center of Innovative Treatments (ped.UCiT) as one of the leading international pediatric clinical trial centers for rare diseases.