To fulfil this aim, the proposals should:

• Establish a network of scientific and technical centres of excellence (new and/or existing laboratories/institutions) complementing each other to enable translational research in ATMPs or other related innovative therapeutic modalities relevant to the future treatment of genetically defined diseases. These scientific and technical centres are expected to provide access and advance translatable, quality-controlled technologies, share data, and build capacity to assist industrial and academic developers of ATMPs.
• Develop tools and methods and define key characteristics of ATMPs, and quality standards that are critical to later stages of development of ATMPs and other related innovative therapeutic modalities, in particular those targeting rare diseases with no approved treatment option. Relevant therapeutic modalities must include appropriate vector systems and innovative modalities such as messenger RNA (mRNA) and nanoparticles (NPs) for therapeutics. Technology areas of interest could include targeted delivery (e.g. methods to target distribution), stability (e.g. methods to increase the stability of RNA), transgene expression, advanced redosing technology approaches/reduced immunogenicity of gene delivery platforms, and other underlying biology relevant to the specific therapeutic modality enabling accelerated translation to clinical development and manufacturing.
• Develop and support the uptake of standardised analytical assays, methods and technological platforms, other non-clinical methods and design strategies.
• Demonstrate the translatability, scalability, and robustness of technologies suitable for the development of subsequent ATMPs and other related innovative therapeutic modalities. This may include process development, mRNA and NPs scale-up and stability, vector production, increasing the throughput of the systematic assessment of the biological and mechanistic features and product characterisation, and ensuring broad accessibility of critical manufacturing materials such as cell lines and producer plasmids.
• Assess the methods and technological platforms developed for their translational and regulatory validity/utility. Define a regulatory pathway to support the fit-for-purpose development of ATMPs, taking into account an evolving regulatory environment and the interplay between all applicable legislation. Ensure early engagement with the regulators so that the methods and data generated support regulatory needs.
• Validate the performance of the methods and technologies developed and demonstrate their higher performance in comparison to existing methods for addressing the bottlenecks in the development and manufacturing cycles of ATMPs and other related innovative therapeutic modalities. In addition, test the functionality of the centres of excellence and demonstrate their capability and performance to support translational research through use cases. The submitted proposals must plan for an open expression of interest / call process to invite third parties, external to the initially established consortium, to submit use cases at least twice during the lifetime of the project.
• Contribute to strengthening the European rare disease ecosystem by engaging all relevant stakeholders, especially patients and patients’ representatives for rare diseases, carers, clinicians, and regulators.
• Define relevant metrics and measure the use of centres of excellence by relevant stakeholders for the development of their assets or novel technological solutions/therapies.
• Define a plan for sustainability beyond the lifetime of the project, including consideration for potential expansion to additional promising technological areas.