Personalized neoepitope-based immunotherapies are considered a promising approach for the targeted treatment of cancer. These therapies are based on tumour-specific neoepitopes which arise through somatic mutations, occurring exclusively in tumour tissue, and can be recognized as foreign by the immune system. Patient-specific selection and GMP-compliant synthesis of these neoepitopes, followed by their use as peptide vaccines, can trigger highly specific immune responses that are intended to selectively inhibit tumour growth.

However, the translational implementation of such individualized peptide vaccines is associated with considerable regulatory challenges. Apart from the traditional compounding of prescriptions in pharmacies, no clearly defined regulatory framework currently exists for individually manufactured, patient-specific drugs. Therapeutic peptide vaccines cannot be clearly assigned to either established drug categories or ATMP regulations. Standardized regulatory approval and GMP concepts are only partially applicable here, as each batch is manufactured exclusively for a single patient, and the drug sequences inevitably differ.

Therefore, robust and reliable guidelines for the development, GMP manufacturing, and further clinical testing of these personalized neoepitope peptide vaccines are to be developed. The focus of the project is the clarification of key regulatory issues regarding platform validation requirements for the highly variable peptide drugs, as well as the necessary preclinical and analytical data for submitting clinical trial applications. Regulatory requirements, GMP guidelines, and the specific characteristics of personalized manufacturing processes will be discussed and finalized with the authorities. The goal is to gain legal clarity early on and reduce regulatory uncertainties without hindering innovation.

In the long term, the project will lay the basis for a regulatory-secure, GMP-compliant platform for the production of personalized neoepitope peptides. The implementation takes place at/and together with the DKFZ and in close clinical cooperation.