Particulates are everywhere, but are they harmful in cell and gene therapies?

Abstract

Cell and gene therapies (CGTs) are a broad class of revolutionary new treatment modalities with enormous potential to impact multiple intractable diseases. In recent years, the sector has reached a breakthrough point, with unprecedented approvals of cell- and gene-based treatments such as adipose-derived mesenchymal stromal cells (Alofisel, manufactured by Takeda Pharma A/S), chimeric antigen receptor T-cell therapies (e.g., Yescarta, Kite Pharma) and gene therapies (e.g., Luxturna, Spark Therapeutics) and lipid nanoparticle (LNP) vaccines against severe acute respiratory syndrome coronavirus 2 virus (Spikevax by Moderna and Comirnaty by Pfizer-BioNTech).