Biotechnology company discovering, developing, and commercializing ex vivo autologous hematopoietic stem cell (HSC) gene therapies for rare, genetic, and other severe diseases
Biotechnology company discovering, developing, and commercializing ex vivo autologous hematopoietic stem cell (HSC) gene therapies for rare, genetic, and other severe diseases. Its approach genetically modifies a patient's own blood stem cells outside the body and reinfuses them to address the underlying cause of disease, with the goal of providing a potentially curative treatment.