Engabexagene cincesparvovec

Engabexagene cincesparvovec is a gene therapy that utilizes a recombinant, non-replicating adeno-associated virus serotype 9 (AAV9) vector to deliver a codon-optimized human gene. Specifically, it encodes the human beta-hexosaminidase subunit beta preprotein, which is relevant for diseases caused by deficiencies in this enzyme[2][3]. The therapy is designed to introduce functional copies of the target gene into patient cells, enabling production of the missing or defective protein. This approach aims to address underlying genetic causes rather than just symptoms. Engabexagene cincesparvovec belongs to the class of adeno-associated viral vector therapies and represents an advanced modality for treating monogenic disorders.