EXG-001

EXG-001 is a non-integrating, temperature-sensitive, non-transmissible Sendai virus vector developed by Elixirgen Therapeutics, Inc.. It is designed to deliver the human Zinc finger and SCAN domain containing protein 4 (ZSCAN4) gene into cells. This vector is used ex vivo to modify autologous CD34+ hematopoietic stem cells, which then become the active component of the cell therapy EXG-34217. The ZSCAN4 protein, once expressed, is responsible for regulating telomere elongation and genome stability, enabling the lengthening of telomeres independently of telomerase. EXG-001's role is crucial in the development of therapies for Telomere Biology Disorders with bone marrow failure.