An advanced gene therapy approach that improves therapeutic efficiency by protecting transgene expressing cells from host immune responses, through modulation of a key immune checkpoint pathway.
The gene therapy market is rapidly expanding, driven by breakthroughs in genetic medicine, increasing approvals of curative therapies, and growing investment from biotech and pharma. Effective gene delivery remains a critical determinant of therapeutic success, with viral vectors (such as AAV and lentivirus) and non-viral systems (including lipid nanoparticles and electroporation) forming the backbone of current and next-generation therapies. As the field matures, improving transgene durability, safety, and immune compatibility has emerged as a key unmet need, creating strong demand for technologies that enhance gene delivery efficiency and enable sustained therapeutic expression across platforms.
Emory researchers have developed the next generation of enhanced multi-transgene delivery system that is equipped with immune modulation to protect transgenic therapeutic cells from host immune activation for a wide range of gene therapy applications. The inventors have engineered a novel variant of immune modulator that is packaged into the delivery capsule and co-administered along with a chosen transgene. This combined delivery using co-expression mediated by fine tuning of expression of promoters creates an “immunologic shield” around transduced cells to allow them to continue to express their desired transgene for a long term. This is a high value technology and can be applied to regenerative medicine, tissue grafting, organ transplantation, and cell-based gene therapies like cancer, autoimmune, and inflammation related diseases.