Tecnologia

AAVantgarde Bio, start-up biotecnologica internazionale con sede a Milano in fase clinica basata su validate piattaforme proprietarie di vettori virali adeno-associati (AAV) per il trasferimento di geni di grosse dimensioni coinvolti in malattie ereditarie della retina. Co-fondata dal Professor Alberto Auricchio e originata dalle attività di ricerca svolte presso il Tigem (Istituto Telethon di Genetica e Medicina) a Napoli, Italia, e supportato da Sofinnova Partners.
Rita Ferla, Fabio Dell’Aquila et al., Efficacy, pharmacokinetics, and safety in the mouse and primate retina of dual AAV vectors for Usher syndrome type 1B. Molecular Therapy: Methods & Clinical Development, March 2023
https://doi.org/10.1016/j.omtm.2023.02.002
Patrizia Tornabene et al., Inclusion of a degron reduces levels of undesired inteins after AAV-mediated protein trans-splicing in the retina. Molecular Therapy: Methods & Clinical Development, December  2021
https://doi.org/10.1016/j.omtm.2021.10.004
Patrizia Tornabene, Ivana Trapani et al., Intein-mediated protein trans-splicing expands adeno-associated virus transfer capacity in the retina. Science Translational Medicine, May 2019.
https://doi.org/10.1126/scitranslmed.aav4523

Pasqualina Colella et al., Efficient gene delivery to the cone-enriched pig retina by dual AAV vectors. Gene Therapy, April 2014.
https://doi.org/10.1038/gt.2014.8

Ivana Trapani, Pasqualina Colella et al., Effective delivery of large genes to the retina by dual AAV vectors. Embo Molecular Medicine, February 2014
https://doi.org/10.1002/emmm.201302948