RNA - Genetherapys
The Project

Research focus

Guided by science and powered by technology, drug treatments for rare genetic diseases begin with the refinement of promising drug candidates. Applying these candidates to cutting-edge applications such as Artificial Intelligence (AI) and Machine Learning (ML) through state-of-the-art computing and storage solutions will provide production-ready and interoperable software tools.
Covering five major areas of human disease, the project concentrates on genetic diseases, cancer, metabolic and cardiovascular diseases, neurodegeneration, and inflammatory and infectious disorders. Translating their scientific findings into tangible products, the project focuses on drug development through Biocomputing, RNA/DNA delivery platforms, pre-clinical development, and regulatory experts that design scalable protocols for industrial production.

Goals of the project

Goals of the project

The ultimate goal of the Center and the community it fosters is to address fundamental science questions and therapeutic applications for the translation of market-ready products. To do so, the Center promotes research & development and the manufacturing of medicines supported by state-of-the-art infrastructures (Gene Therapy Center , RNA Production Platform) and the advancement of a highly trained workforce (PharmaTech AcademyNational PhD).

The Fundamental Science Questions

The fundamental science questions

Known as a mediator between the information encoded in DNA and its translation into proteins, the successful exploitation of RNA became evident with the COVID-19 vaccines as messenger RNA instructed human cells to produce viral proteins. Over the last 30 years, we have discovered other types of RNA with cellular roles spanning from gene silencing to protein regulation. Indeed, alterations in RNAs that do not code for proteins but rather finely control their expression underlie many human diseases leading to gene-silencing RNAs as targeted drugs. The National Center addresses the fundamental science questions for a better understanding of the interactions of RNAs and their therapeutic applications.

Therapeutic applications

The therapeutic applications

RNA is the prototype answer to the fundamental science question in the search for personalized and flexible drugs. Capable of responding to the individual path of a disease, such as the sub-type of cancer or the variant of a virus, customizing RNA is the future of personalized medicine. RNAs are flexible because their mode of action controls their molecular details. For example, in the fight against cancer, RNA silencing can stifle the gene that promotes it or can boost a natural reaction from the immune system. Paying specific attention to translatability (state-of-the-art infrastructures to manufacture RNAs and their formulations), the Center fosters personalized applications in cancer, neurodegenerative, and inflammatory diseases.

Program

The National Center for Gene Therapy and Drugs based on RNA Technology secures funding under Investment 1.4 of the National Recovery and Resilience Plan (PNRR). The allocation of €320,036,606.03 will focus on supporting Key Enabling Technologies related to the “Development of Gene Therapy and Drugs based on RNA Technology.” Managed by the National Center Foundation – Gene Therapy and Drugs based on RNA Technology and spearheaded by the University of Padua, the 36-month program began on the 1st of November 2022. The National Center (NC RNA & Gene Therapy) unites 46 organizations, including universities, research institutes, and private companies, fostering national and international innovation.

Organized into ten distinct areas known as Spokes, each part of the research program focuses on a specific subject and includes the involvement of affiliates and third parties identified through open and cascading calls. The research efforts recognized as crucial for advancing knowledge in Gene Therapy and RNA Technology-based Drugs are defined within 5 Vertical Spokes and 5 Horizontal Spokes.

Designed to leverage a synergistic interaction between academia and private partners, the collaborative nature of research activities held within each Spoke facilitates a reciprocal exchange of expertise and competence. This interconnection is strategically aligned to link the technological advancements of Horizontal Spokes by providing the tools and the know-how relative to the five key areas of human diseases within Vertical Spokes. This collaboration draws upon the strengths of multinational entities, Italian biomedical and pharmaceutical companies, the largest Italian bank, and several biotech companies active in the rapidly expanding field of RNA therapeutics and gene therapy. This cohesive interconnection plays a crucial role in translating proof-of-concept study results from academic laboratories and identifying opportunities to transfer knowledge from academia to private stakeholders.

Vertical Spokes

The primary objective of Vertical Spokes is to identify and develop the most promising candidate targets for RNA-based drugs across the following five key areas of human diseases: genetic diseases, cancer, metabolic and cardiovascular conditions, neurodegenerative disorders, and inflammatory/infectious ailments.

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Horizontal Spokes

The primary objective of Horizontal Spokes is to develop and share their technological expertise for designing, delivering, and manufacturing gene therapy products and RNA drugs. The anticipated outcomes rest on three foundational pillars: the identification and validation of targets for both RNA-based drugs and gene therapy/genome editing approaches; the technological advancement in Key Enabling Technologies (KET); and the establishment of core facilities for the production of RNA and gene therapy products.

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