zaftuclenegene piruparvovec

Zaftuclenegene piruparvovec is a recombinant, self-complementary (dimeric), non-replicating adeno-associated virus serotype 9 (scAAV9) vector-based gene therapy. It encodes a codon-optimized human palmitoyl-protein thioesterase 1 (PPT1) gene. The therapy is designed to deliver the functional PPT1 gene to patients with deficiencies, aiming to restore enzyme activity and address the underlying cause of disease. This modality leverages AAV9’s ability to cross the blood-brain barrier and efficiently transduce cells in the central nervous system[2]. Zaftuclenegene piruparvovec is under investigation for use in genetic disorders related to PPT1 deficiency.