Source: Healio News
The FDA granted orphan drug designation to CT103A, a chimeric antigen receptor T-cell therapy in development for adults with relapsed or refractory multiple myeloma.
CT103A (IASO Biotherapeutics/Innovent Biologics) is an autologous, gene-edited CAR T-cell therapy that targets the B-cell maturation antigen on the surface of cancer cells.
The agent contains a unique fully human single-chain variable fragment sequence and a 4-1BB costimulatory and CD3-zeta domain encoded within the lentiviral vector.
“[The granting of orphan drug designation] to CT103A is of great significance to patients