RNA - Genetherapys

BioPharma Encapsulated: Unveiling the Spokes of the National Center for Gene Therapy and Drugs based on RNA Technology

If you have not already, we recommend reviewing previous publications where we explain the roles and functions of DNA and RNA and how their various alterations manifest disease.

This article will take a deep dive into the Hub & Spoke organizational structure of the National Center to clarify who and what the Spokes are, how their Vertical and Horizontal alignment interconnects, and what role the Hub plays in its balance.

Why bring together entities as diverse as universities and private companies?

Research projects conducted at universities are crucial to the scientific community, as most discoveries occur in their laboratories. Companies often use these spaces and equipment, specifically those areas defined by their R&D (Research and Development), while both conduct research, the approach and the purpose of their studies vary within different mindsets. Companies focus on development, as, in addition to molecular discoveries, they deal with creating and developing drugs for patients. Similarities end here, as university laboratories consider all mechanisms of the functioning of each single drug as they study molecules using various approaches to minimize side effects. Ultimately, despite their differences, universities and companies must collaborate and support each other by analyzing different aspects of the same topic. 

 

After clarifying the research carried out at companies compared to those at universities, let us move on to the project.  Structurally organized by Spokes and a Hub, such as those found on a bike wheel, the project follows ten different research areas divided into Vertical and Horizontal subgroups. Vertical Spokes seek to identify WHAT research will take place, while Horizontal Spokes focus on HOW to exploit the findings that emerge from the Vertical Spokes. Each Spoke collaborates with affiliated bodies that work on the research project dictated by the relevant Spoke. With many affiliated bodies, the numbers vary from Spoke to Spoke. For each Spoke leader, the number of affiliates falls between a minimum of seven and a maximum of 15.

Determined to advance knowledge on gene therapy and RNA technology, the Italian National Recovery and Resilience Plan (PNRR) allocated funding for the collaboration of a series of public and private bodies working towards the same objective but with different methods and different visions. This undertaking offers strength in unity and will bring together various skills and competencies, which will lead to something great.

The goal is to merge the brightest minds, each with different academic and professional backgrounds, to improve the quality of life of others.

In addition to the Spokes, it is important to highlight the Hub’s central role in coordinating this complex project.

Officially entitled, the National Center for Gene Therapy and Drugs based on RNA Technology, the Hub began operating on 22 November 2022.  With an investment of €320,036,606.03, the Hub allocates these funds towards the development of Key Enabling Technologies related to gene therapy and RNA-based therapeutics.

The Hub represents the heart of the project, acting as the managerial interface, not only with the Spokes but with ministerial representatives, and coordinates their actions.

Actively involved in research projects for young researchers, the Hub also promotes the creation of several infrastructures, including the RNA Facility in Naples and the Gene Therapy Center. The Hub also promotes the technological transfer of discoveries through supporting spin-offs and start-ups, while investing in National PhD and PharmaTech Academy programs, which will train highly specialized professional figures with transversal skills.

Therefore, by presenting the various actors of the project we share how this complex machine functions, with its Spoke focused on implementing the research and project directly with universities and pharmaceutical laboratories, and a Hub that coordinates their efforts. A complex machine that will impact the life-long learning of a new generation and the goals found along their educational career paths to compete in a growing field. 

Next week we will learn more about the characteristics of each Spoke. Stay tuned to learn more about the groundbreaking research that Spoke 1 is carrying out on its relevant topics: genetic diseases of the skin, blood, eye, storage diseases, kidney, and neuromuscular and neurometabolic disorders.

 

Written by

PharmaTech Academy student Laura De Cantis

and the Foundation’s editorial staff

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