Red blood cell extracellular vesicles / Carmine Therap, Cystic Fibrosis Foundation - LARVOL DELTA

Unlocking extracellular vesicles-mediated efficient DNA delivery for long-lasting transgene expression. (PubMed, Mol Ther) - "Incorporating heavy and light chain antibody constructs into RBCEVs enables sustained expression of Herceptin antibodies in circulation. Further utilization of cell-specific promoters enhances transgene expression and concomitantly reduces undesired immune responses. These results highlight RBCEVs as a safe, scalable, and versatile DNA delivery platform for achieving sustained transgene expression in gene therapy."

Journal • Gene Therapies

Extracellular Vesicles Administered via Intrathecal Injection Mediate Safe Delivery of Nucleic Acids to the Central Nervous System for Gene Therapy. (PubMed, J Extracell Vesicles) - "Furthermore, experiments conducted in both mouse and non-human primate models indicate that intrathecal injection of plasmid-loaded RBCEVs do not lead to any systemic or local acute toxicity. In summary, our findings illustrate the potential of the RBCEV-based platform as a viable and safe approach for nucleic acid delivery to the CNS, facilitating further development of gene therapy for neurological disorders."

Journal • CNS Disorders • Gene Therapies

IL-12 minicircle delivery via extracellular vesicles as immunotherapy for bladder cancer. (PubMed, Cell Prolif) - "Intratumoral delivery of Il-12 plasmid-loaded RBCEVs suppressed bladder cancer tumour growth, stimulated immune responses and promoted immune cell infiltration. In conclusion, our study demonstrates the promising potential of RBCEVs as an effective, safe and redosable nucleic acid drug delivery platform for IL-12."

IO biomarker • Journal • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor • IL12A