Topline results from Phase 1/2 AAV gene therapy (LX1001) in APOE4/4 homozygotes with Alzheimer’s disease

Abstract

Background: APOE4 homozygotes with Alzheimer’s have faster rates of cognitive decline and become symptomatic approximately a decade earlier. APOE2 is a protective variant with reduced likelihood of developing AD and a slower rate of decline. LX1001 is an adeno‐associated viral vector investigational gene therapy (AAVrh.

The paper