close

Location

345 Park Avenue South, 6th Floor
New York, NY 10010
(212) 547-9879

CNS programs

Advancing Gene Therapy in Monogenic Diseases

LEXEO aims to apply the transformational science of gene therapy to address some of the most devastating central nervous system diseases affecting both rare and prevalent patient populations. We are developing a pipeline of CNS focused gene therapies that includes a portfolio of approaches to treat the genetics underlying Alzheimer’s disease.

LX1001 / LX1020 / LX1021

Alzheimer’s

Our current gene therapy programs under development for the treatment of Alzheimer’s disease are:

  • LX1001: a gene therapy candidate that is designed to express the protective APOE2 protein
    • in the CNS of APOE4 homozygous patients.
  • LX1021: a gene therapy candidate that is designed to express the Christchurch-modified
    • APOE2 protein in the CNS of APOE4 homozygous patients. The Christchurch mutation has been observed to protect patients against Alzheimer’s disease even in the presence of significant amyloid pathology.
  • LX1020: a gene therapy candidate that is designed to express the protective APOE2 protein in the CNS of APOE4 homozygous patients, while concurrently delivering miRNA to suppress the expression of the APOE4 protein.
We are developing LX1001 to target homozygous APOE4-associated Alzheimer’s disease patients, the highest risk population to develop Alzheimer’s disease and the sub-group we believe will be the most likely to demonstrate a potential treatment effect from APOE2-based gene therapy.

learn more