Orchestrating Global Cell and Gene Therapy Translation Building Consensus for the Path Forward
Human trials of exosome therapeutics mark the arrival of true clinical translation. Understanding manufacturing and regulatory challenges is critical to ensure a smooth translational path forward.
"Scientific rationales and generation of evidence necessary for proof-of-concept are key considerations in developing human clinical trials. Interdisciplinary collaboration to translate preclinical data will inform successful development of exosomes into therapeutics"
"It is largely unclear how the EV field should translate its wealth of preclinical data to clinical trials. To undertake this task effectively, it will be critical to seek consensus on issues such as translating effective dosage from animal to human studies, quantifying EV drug preparation, investigating toxicity, and investigating the MoA to validate in vitro potency assays"