RNA - Genetherapys
Infrastructures

In the framework of the Research Program, the National Center promotes the creation of two infrastructures to the benefit of all the National Center members and the broader Italian scientific community. The Gene Therapy Center will take advantage of pre-existing facilities located at Bambino Gesù Children’s Hospital in Rome (OPBG), San Raffaele Telethon Institute for Gene Therapy (SR-TIGET) and the Tettamanti Foundation. The RNA Production Platform will be implemented in a dedicated space at the University of Naples Federico II.

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Gene Therapy Center RNA Production Platform
Gene Therapy Center

Gene Therapy Center

While prioritizing the needs of patients, the Gene Therapy Center will also implement clinical applications for new technologies.
Hosted and managed by Bambino Gesù Children’s Hospital in Rome (OPBG), San Raffaele Telethon Institute for Gene Therapy (SR-TIGET) and Tettamanti Foundation, the facilities available to the Gene Therapy Center hold current Good Manufacturing Practice (GMP) requirements needed to engage in the gene modification of somatic cells.
Attentive to sustainability efforts, renovated facilities will scale up to GMP-compatible processes required for innovative drug products and subsequently early-stage clinical trials. The Gene Therapy Center will improve the clinical translation and create a service structure needed to bring gene therapy products to the market. The Gene Therapy Center will occupy the manufacturing, training, and consultation of gene therapy products, thus contributing to advancements in the field.

RNA production platform

RNA Production Platform

The RNA Production Platform will serve National Center research groups as a space devoted to RNA synthesis and formulation. The state-of-the-art facilities within the RNA Production Platform ensure a manufacturing process that is both agile and capable of enduring robustness and reproducibility during product production. Providing Italy with its first National Platform for high-quality RNA production, the realization of an RNA Production Platform for the National Center represents the essential support needed for the industrialization of R&D advancements sought out by disease-oriented Vertical Spokes. Additionally, for those researching RNA synthesis, formulation, and delivery of pre-clinical in vitro and in vivo testing of RNA products, the facilities also support the needs of the technology-oriented Horizontal Spokes.