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LX1021 is a gene therapy candidate developed by Lexeo Therapeutics for the treatment of Alzheimer's disease in patients who are homozygous for the APOE4 allele. The therapy is designed to deliver a Christchurch mutation-modified APOE2 allele into the central nervous system, aiming to express a neuroprotective form of the APOE2 protein. This modified protein is intended to counteract the high genetic risk conferred by two copies of APOE4, which significantly increases susceptibility and accelerates progression of Alzheimer's disease. Preclinical data suggest that LX1021 may have an enhanced effect on reducing tau pathology compared to earlier gene therapies targeting APOE2 alone[1][2][4][5]. The program remains at an early (preclinical) stage.
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