RNA - Genetherapys

National Center for Gene Therapy

and Drugs based on RNA Technology

 

By sharing knowledge and resources, we can accelerate the development of cutting-edge RNA and gene therapies

 

 

 

What is RNA and gene therapy?

As an interdisciplinary field, which includes molecular biology, genetics, biotechnology, pharmaceutics, pharmacology, and clinical medicine, research activities based on ribonucleic acid (RNA) and gene therapy aim to create a step change in healthcare through treatments that are:

  • Personalized – based on the molecular profile of a specific individual (genetic – DNA or transcriptomic – RNA)
  • Precise – targeting the molecular roots of a disease (e.g. a neurodegenerative pathway), thus minimizing effects on physiological mechanisms
  • Flexible – adapting to the changing pattern of disease (e.g. a virus or a tumor becoming therapy-resistant)
  • Variably lasting – duration tunable from days (e.g. antigen production caused by mRNA vaccines) to lifelong results (e.g. through DNA modification).
RNA gene theraphy

What is the National Center for RNA & Gene Therapy?

The National Center for Gene Therapy and Drugs based on RNA Technology, funded by the EU National Recovery and Resilience Plan (NRRP Mission 4 – Component 2, Investment 1.4), and spearheaded by the University of Padua, brings together 46 Italian and international partners from the public, private, and corporate sectors and organizes them within a 36-month research program.

Clinical-laboratory

Activities of the research program aim to field a better understanding of diseased molecular mechanisms and to develop therapies for them. Its overarching strategic goals are to support the efficacy and sustainability of the healthcare system, within the framework of a competitive knowledge-based economy.

The National Center is home to a large number of research projects structured along the Hub and Spoke organizational model, which allows for the interconnection of Vertical Spokes and Horizontal Spokes. Vertical Spokes aim to identify the most promising candidate targets for RNA-based drugs, while Horizontal Spokes aim to build and disseminate the technological know-how that designs, delivers, and produces gene therapy products and RNA drugs. The interconnection will expand RNA-based pharmacology and gene therapy and develop new drugs for precision medicine. Further, the National Center will focus on the creation/strengthening of cutting-edge infrastructures and the promotion of high-tech entrepreneurial and educational initiatives.

National PhD

The RNA Therapeutics and Gene Therapy National PhD program aims to train highly qualified researchers with the scientific expertise and autonomous research skills needed to address the challenges of RNA-based drugs and gene therapy.

As a comprehensive three-year doctoral degree program, guided by the Department of Pharmacy at the University of Naples Federico II, the program involves ten universities and two research centers across Italy.

National-PhD
PharmaTech Academy

PharmaTech Academy

PharmaTech Academy is a training initiative of the National Center led by the University of Napoli Federico II, Department of Pharmacy. Its main objective is to train highly qualified professionals to operate across all phases of RNA-based drug development, thanks to a practical and professional educational approach based on the concept of “learning by doing”.

Built on a close collaboration between universities and private companies participating in the National Center, the Academy aims to expand the skills acquired during degree programs by offering hands-on experience and direct contact with the pharmaceutical industry, thus contributing to the creation of specialized figures in the field of RNA gene therapy in Italy.