The Food and Drug Administration (FDA) has granted Fast Track designation to LX1001 (Lexeo Therapeutics) for the treatment of apolipoprotein E4 (APOE4) associated Alzheimer disease.

Evidence has suggested that the inheritance of APOE4 gene results in more frequent and earlier onset of Alzheimer disease, while the inheritance of APOE2 gene has the reverse effect. The investigational adeno-associated virus mediated gene therapy, LX1001, is being developed to deliver the protective APOE2 gene to the central nervous system. 

The Company is currently conducting an open-label, dose-ranging phase 1 trial ( Identifier: NCT03634007), which will assess the safety and tolerability of LX1001 for the treatment of patients with early-stage Alzheimer disease with mild cognitive impairment who are APOE4 homozygous. Initial trial data is expected in the second half of 2021.

Continue Reading

R. Nolan Townsend, CEO of Lexeo Therapeutics, commented, “While other treatments in development focus on the pathogenesis of Alzheimer disease, LX1001 is the first investigational gene therapy designed to correct the underlying genetics of the disease thereby addressing the most significant risk factor for developing Alzheimer’s disease.”

The FDA’s Fast Track designation allows for expedited review of therapies that are meant to treat serious or life-threatening conditions. Generally, the designation is granted to drugs that are expected to have an impact on factors such as survival and daily functioning.


Lexeo Therapeutics announces FDA Fast Track designation granted to LX1001 for the treatment of APOE4 associated Alzheimer’s disease. [press release]. New York, NY: Lexeo Therapeutics; April 20, 2021.

This article originally appeared on MPR