RNA - Genetherapys

BioPharma encapsulated – Spoke 8

organs-on-a-chip - 1

Spoke 8 – Platforms for RNA/DNA Delivery

Padua, 17 October 2024 – Hindered by its poor stability in the organism, the limitations of using RNA for therapeutic purposes are due to its inability to reach targeted sites and effectively penetrate cells.

The 100 researchers working in Spoke 8, spread across 10 Italian institutions and 5 internationally known pharmaceutical companies, hold consolidated experience in drug delivery technologies as they collaborate on the development of new RNA delivery platforms capable of navigating from the site of administration to the cell where the RNA must act upon while protecting it from degradation. Aimed at building an extensive library of advanced delivery platforms using both non-viral and viral technologies, Spoke 8 researchers focus on developing innovative lipid and polymeric nanoparticles, viral vectors, and vesicles that facilitate precise delivery of nucleic acids to target cell populations and diseased areas within the body.

To evaluate toxicity and biological activities of delivery platforms, with particular reference to solid tumors, researchers focus on developing alternative biological models such as organoids and organ-on-chip while exploiting zebrafish’s cost-effectiveness and optical transparency. Omics sciences and advanced imaging allow Spoke 8 to integrate in vitro studies and uncover the mechanisms by which delivery systems work.
New technologies for producing RNA-based nanoplatforms, such as microfluidics and supercritical fluids, are in development. The goal is to scale up laboratory production in Italy at industrial volumes while ensuring the stability and storage of a final product.

Implementing production under Good Manufacturing Practices (GMP) guarantees a high-quality profile for prototypes, which is crucial for subsequent preclinical studies. Platforms that meet the preliminary criteria of quality, efficacy, and safety ensure that new therapeutic RNAs developed by Spokes 1 – 5 can move into clinical trials.

Spoke 8 researchers are also interested in developing AI-based solutions to enable faster development of optimized delivery prototypes.

Spoke 8 affiliates include Antares Vision Group, Chiesi Group, National Research Council of Italy (CNR), Dompè Pharmaceuticals, Italian Institute of Technology, Stevanato Group, Takis Biotech, Sanofi, University of Milan (UNIMI), University of Milano-Bicocca (UNIMIB), University of Naples Federico II (UNINA), University of Palermo (UNIPA), University of Padua (UNIPD), University of Pisa (UNIPI), University of Salerno (UNISA), and University of Turin (UNITO).

Foundation staff writer Ellen Jane Corcoran

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