DYD-701

DYD-701 (also known as LCAT-GMAC) is an autologous ex vivo gene therapy and cell therapy being developed by DyDo Pharma in collaboration with CellGenTech for the treatment of familial lecithin-cholesterol acyltransferase (LCAT) deficiency (FLD). The therapy involves extracting cutaneous fat from the patient, isolating adipocytes, and genetically modifying them using viral vectors to express the functional LCAT enzyme. These gene-modified adipocytes (GMAC) are then transplanted back into the patient to provide a continuous source of LCAT, aiming to restore enzyme activity and manage the severe metabolic consequences of FLD, such as corneal opacity, hemolytic anemia, and progressive renal failure. The development has been supported by the Japan Agency for Medical Research and Development (AMED).

Other names

LCAT pre-transgenic adipocytegenetically modified adipocytes expressing LCAT