Technology
AAVantgarde Bio platforms are focused on broadening the application of AAV based therapies by addressing the limitations of single AAV therapies cargo capacity.~1,200 ocular and non ocular human coding sequences are above standard AAV carrying capacity and could be leveraged using our platform technology.
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
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
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
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
https://doi.org/10.1002/emmm.201302948