Shots:

• The EMA has accepted the marketing authorization application for RP-L102 for the treatment of Fanconi anemia. The submission of its BLA to the US FDA is anticipated in H1’24   
• The MAA was supported by results from the P-I/II study of RP-L102 showing sustained genetic & phenotypic correction as well as hematologic stabilization for 42mos. without any new safety outcomes. No symptoms were observed for bone marrow dysplasia, clonal dominance or insertional mutagenesis
• RP-L102 is a gene therapy composed of autologous hematopoietic stem cells that were genetically modified using a lentiviral (LV) vector to carry a functional FANCA gene

Ref: Rocket | Image: Rocket

Related News:- Rocket Reports the US FDA Acceptance of BLA for RP-L201 (marnetegragene autotemcel) to Treat Severe Leukocyte Adhesion Deficiency-I

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