AM805
Investigational AAV9 gene therapyAn investigational AAV9-mediated gene therapy designed as a one-time treatment for Alzheimer's disease, delivering protective protein/cathepsin A (PPCA) — a lysosomal carboxypeptidase — directly to the brain to degrade amyloid-beta (Abeta42).
- AAV9 vector delivering the PPCA enzyme to the brain
- Catalytic protease mechanism that cleaves amyloid-beta and prevents oligomer formation
- Cross-correcting mechanism: PPCA secreted by transduced cells is absorbed by neighbouring cells for widespread clearance
- Reduces both intracellular and extracellular amyloid-beta in preclinical models