RNA - Genetherapys
Gene Therapy Center

The Gene Therapy Center provides support to the needs of patients while fostering the dissemination of clinical applications for new technologies.
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, which both follow current Good Manufacturing Practices (GMP) that authorize them for the gene modification of somatic cells.

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Gene-Therapy-Center

Attentive to sustainability efforts as described in Italy’s National Recovery and Resilience Plan, the Gene Therapy Center will undergo refurbishments that ensure its facilities scale up to GMP-compatible processes of the innovative drug products to be tested subsequently in early-stage clinical trials.
Determined to fulfill its objectives, the Gene Therapy Center focuses on improving the clinical translation of Gene Therapy (GT) products and creating a service structure for the production of GT products.

To improve the clinical translation of GT products, new gene therapy applications will move rapidly from lab-grade protocols through optimization and scale-up to GMP-compatible processes ready for tech transfer to an authorized GMP cell factory and adequate for early-stage clinical trials.
Given Italy’s current research activities, the acceleration ensured by the National Center will provide highly efficient infrastructures operating in the final step to guarantee the rapid transfer of scientific advancements to the benefit of patients.
Moreover, the Gene Therapy Center will be available to the other members of the National Center, which will greatly increase the accessibility of these innovative treatments while reducing inter-regional differences and mitigating sanitary migration.
By creating a service structure for the production of the GT products, the Gene Therapy Center will act as a service and training hub. It will reduce the heterogeneity of products, such as gene-modified cells and vectors, and establish standardized procedures and common best practices. Designing a service structure for the production of GT products with a focus on standardization and common best practices is crucial for ensuring quality and reliability in this field.
The Gene Therapy Center will serve as a focal point for the manufacturing, training, and consultation of gene therapy products, thus contributing to advancements in the field.