FV-coNF1

FV-coNF1 is an investigational gene therapy developed by researchers at the Mayo Clinic for the treatment of Neurofibromatosis type 1 (NF1) and associated tumors, such as malignant peripheral nerve sheath tumors (MPNSTs) and plexiform neurofibromas (PNFs). It utilizes a foamy viral (FV) vector, which is chosen for its large cargo capacity and favorable integration profile, to deliver a full-length, codon-optimized version of the NF1 transcript 2 isoform (coNF1). By restoring the expression of functional neurofibromin in affected Schwann-cell lineages, FV-coNF1 aims to suppress the hyperactivation of the MAPK pathway and inhibit tumor growth. Preclinical studies in xenograft models have demonstrated significant tumor regression and eradication following intratumoral administration, with the codon-optimized transcript localizing appropriately and reducing aberrant Ras activity.