Amlogenyx

Amlogenyx is a Novato, California biotechnology company developing gene therapies that reduce amyloid accumulation in the brain in order to prevent and treat Alzheimer's disease and other amyloid diseases. It was spun out of Ultragenyx Pharmaceutical (NASDAQ: RARE), which retains majority ownership, and closed a $14M seed round backed by Ultragenyx and GordonMD Global Investments. Its lead candidate AM805 is an investigational AAV9 gene therapy that delivers protective protein/cathepsin A (PPCA), a lysosomal enzyme that degrades amyloid-beta. The company reported positive preclinical data in March and May 2026, plans to submit an IND later in 2026, and anticipates Phase 1/2 studies in 2027.

Instead of clearing amyloid with monoclonal antibodies, AM805 uses a catalytic enzyme delivered once by gene therapy. PPCA secreted by transduced cells is taken up by neighbouring cells — a cross-correcting mechanism that degrades both intracellular and extracellular amyloid-beta, with preclinical amyloid reductions the company reports as comparable to or exceeding approved Alzheimer's antibodies.