RP-L301

RP-L301 is an ex vivo gene therapy designed for the treatment of pyruvate kinase deficiency (PKD), a rare, autosomal recessive, non-spherocytic hemolytic anemia caused by mutations in the PKLR gene. The therapy consists of autologous CD34+ hematopoietic stem cells harvested from the patient and transduced with a lentiviral vector carrying a codon-optimized red cell pyruvate kinase (coRPK) gene. After myeloablative conditioning, these genetically corrected cells are infused back into the patient to restore functional pyruvate kinase activity in red blood cells. This approach aims to correct the underlying glycolytic defect responsible for increased red cell destruction and chronic anemia in PKD patients. Clinical studies have shown that RP-L301 can lead to sustained increases in hemoglobin levels and eliminate transfusion requirements, with favorable safety outcomes observed so far[1][5][6]. The drug is being developed by Rocket Pharmaceuticals under license from Centro de Investigacion Biomedica en Red de Enfermedades Raras (CIBERER), CIEMAT, and Instituto de Investigacion Sanitaria Fundacion Jimenez Diaz[5].

Brand names

Merilen

Other names

LV RP-L301