Expression Therapeutics has developed several engineering platforms to enable the development of novel gene therapies. Our ‘expression’ technology portfolio includes target cell-specific codon optimization, tissue-directed synthetic transcriptional regulatory elements, and a protein enhancement technology based on ancestral sequence reconstruction (ASR). Although each technology platform involves independent design principles, they can be integrated to achieve optimal safety and efficacy.   

The key to effective ex vivo gene therapy is effective selection and manufacturing of the cell product. In the case of inherited genetic disorders where lifelong production of therapeutic protein is required, our hematopoietic stem and progenitor (CD34+) cell platform is optimal as these cells have the capacity to self-renew and re-populate the blood cell compartment for life. However, in the case of cancer therapy, lifelong survival of the cellular gene therapy may not be optimal due to side effects related to non-cancer cell toxicity. For this reason, Expression Therapeutics has developed a gamma delta (γδ) T cell therapy platform that can provide potent short-term anti-cancer therapeutic benefit without the risk of long-term persistence and toxicity to otherwise healthy tissue.