PNRR - Life Science Hub for Advanced Therapies – LSH-TA National integrated system for implementing academic gene therapy/genome editing approaches aimed at treating inherited disorders

Scritto il 15 Novembre 2023.

To create a national integrated system for implementing academic gene therapy/genome editing approaches aimed at treating inherited disorders.

ATMPs are the ultimate frontier of precision and personalized medicine, where the diseases are tackled, often at molecular level, through treatments based on the infusion of ex vivo cultured/manipulated, sometimes genetically modified, somatic cells. ATMPs also represent a fast-growing field in terms of innovation, investment and business opportunities. The aim of this proposal is to promote the full valorization and deployment of national translational research on ATMPs. The approach to achieve this goal is the creation of a national consortium for developing, validating, and manufacturing clinical grade ATMPs. This consortium is based on a Hub and spoke model that will enable the set-up of different robust and effective translational platforms for the benefit of various potential stakeholders, mainly at national but also international level. This consortium will support the Italian researchers of the participating institutions in advancing the development of their products from the laboratory through the preclinical development to the clinical stage.

The LSH-TA is willing to create a largely shared and homogeneous qualification of the different institutions in the clinical use of ATMPs, complementing the availability of commercially approved products with those generated through the creation of this platform and standardizing the approaches used for monitoring ATMPs safety, persistence and clinical efficacy. In addition, the LSH-TA has also the ambition to attract investments by biotech and pharma industries for reaching the goal of being self-sustained at the end of the initial 4 years supported by MoH.