摘要：Advanced therapy medicinal products (ATMP) can offer new, effective therapeutic options
for the treatment of severe illnesses, including cancer, neurodegenerative and cardiovascular
diseases. Translation of advanced therapies to the clinic has been slow despite significant academic
research from academia and foundations. The implementation of 2001/20 Directive in Italy established
that the development of an ATMP should follow the GXP rules – good manufacturing practice
(GMP) for production, good laboratory practice (GLP) for non clinical safety studies and good
clinical practice (GCP) for clinical trials. The high costs of GCP application and the needs for GMP
facilities are perceived as the most important bottlenecks for the development of ATMP. Here it is
pointed out that a strategic cooperation between different actors (academia, industry and experts
in regulatory issues) is strongly needed. In particular, it is highlighted that the Istituto Superiore di
Sanità, as the competent authority for the authorization of Phase I clinical trials, has a specific responsibility
in fostering the translation of safe and effective therapies for human diseases.
