AAV.CPP.16-ASAH1

AAV.CPP.16-ASAH1 is an adeno-associated virus (AAV) gene therapy candidate designed for the treatment of acid ceramidase deficiency, a lysosomal storage disorder that manifests as Farber disease (FD) and spinal muscular atrophy with progressive myoclonic epilepsy (SMA-PME). The therapy utilizes an engineered AAV9 capsid, designated AAV.CPP.16, to deliver a functional human ASAH1 gene. By restoring acid ceramidase activity, the treatment aims to reduce the toxic accumulation of ceramides, thereby preserving neuromotor function and mitigating neurodegeneration. Preclinical studies in mouse models have demonstrated dose-dependent survival benefits, improved growth, and preservation of grip strength, while non-human primate studies have shown a favorable safety profile following intracerebroventricular administration.