LX1001

LX1001 is an investigational, one-time gene therapy designed to treat APOE4-homozygous Alzheimer’s disease by delivering a protective APOE2 gene into the central nervous system (CNS) using an adeno-associated virus serotype rh10 (AAVrh10) vector. The therapy aims to express the neuroprotective APOE2 protein in the CNS of patients who have two copies of the high-risk APOE4 allele, with the goal of halting or slowing Alzheimer’s disease progression. Clinical data show dose-dependent increases in CSF APOE2 expression, stabilization of amyloid pathology, and reductions in tau biomarkers associated with Alzheimer’s disease. The treatment has demonstrated a favorable safety profile and is being evaluated in ongoing Phase 1/2 clinical trials for patients with mild cognitive impairment or mild-to-moderate dementia due to Alzheimer's disease[1][2][3][4][5][6][7][8][9].

Other names

AAVrh.10hAPOE2LX-1001LX1001LX 1001