RP-L101

RP-L101 is an ex vivo lentiviral vector-based gene therapy candidate developed for the treatment of Fanconi anemia, most specifically severe forms such as FA complementation group A, by transducing autologous hematopoietic stem and progenitor cells with a functional copy of the defective gene to restore DNA repair capacity and improve hematopoiesis. It was originally developed at Fred Hutchinson Cancer Research Center and subsequently in‑licensed and advanced by Rocket Pharmaceuticals into early clinical development, where modified CD34+ cells are collected, gene-corrected using a lentiviral vector, and reinfused following conditioning, aiming to provide durable engraftment of corrected stem cells, reduce bone marrow failure, and decrease transfusion requirements.[5][9][11]