AAV6.2FF-SFTPB

AAV6.2FF-SFTPB is an adeno-associated virus (AAV) gene therapy candidate designed for the treatment of surfactant protein B (SPB) deficiency, a rare and fatal autosomal recessive disorder that causes neonatal respiratory distress syndrome. The therapy employs a modified AAV6 capsid, designated AAV6.2FF, to deliver a functional copy of the SFTPB gene directly to alveolar type 2 (AT2) cells in the lungs. By restoring the expression of surfactant protein B and the proper structure of lamellar bodies, the drug aims to improve lung function and prolong survival. Preclinical studies have also investigated the co-administration of AAV6.2FF-SFTPB with small-molecule vector potentiators (VEPO™) to enhance transduction efficiency and reduce the required viral dose.