PGK-FANCA-Wpre* transduced CD34+ cells

PGK-FANCA-Wpre* transduced CD34+ cells is an investigational autologous gene therapy developed for the treatment of Fanconi anemia subtype A (FA-A). The therapy involves the ex vivo genetic modification of a patient's own CD34+ hematopoietic stem and progenitor cells (HSPCs) using a lentiviral vector (PGK-FANCA-Wpre*) that encodes a functional copy of the *FANCA* gene. This gene is mutated in the majority of Fanconi anemia cases, leading to defective DNA repair and progressive bone marrow failure. The modified cells are re-infused into the patient without the need for prior cytotoxic conditioning, as the gene-corrected cells possess a natural selective advantage in the bone marrow environment. The therapy was initially developed through a collaboration of Spanish research institutions, including CIEMAT and the Hospital Infantil Universitario Niño Jesús, and was evaluated in the Phase I/II FANCOLEN-1 clinical trial. Rocket Pharmaceuticals later acquired the rights to further develop this technology under the designation RP-L102.

Brand names

Fancalen

Other names

Autologous CD34+ cells transduced with a lentiviral vector carrying the FANCA geneFANCA-transduced CD34+ cellsFANCOLEN-1FANCOLEN1FANCOLEN 1