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.