Gene therapy targets Alzheimer's in High-Risk group

NCT ID NCT03634007

Completed Disease control Sponsor: Lexeo Therapeutics Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This early-stage trial tested a gene therapy called LX1001 in 15 people with Alzheimer's who have two copies of the APOE4 gene, a high-risk form. The therapy delivers a healthy APOE2 gene to try to change the course of the disease. The main goal was to check safety and side effects over 52 weeks.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: LX1001 (a gene therapy that delivers a healthy APOE2 gene)
What this could lead to: If it works, this could point toward a treatment that slows or modifies Alzheimer's progression in people with two copies of the APOE4 gene.
What could go wrong: This is a very early, small safety trial with only 15 participants. It may not show clear benefit, and gene therapy carries risks like immune reactions or inflammation.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

Alzheimer disease early-onset autosomal dominant Alzheimer disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

ALZHEIMER DISEASE EARLY-ONSET ALZHEIMER DISEASE

Contacts and locations

Locations

Duke University

Durham, North Carolina, 27708, United States
K2 Medical Research

Maitland, Florida, 32751, United States
PPD- Orlando Research Unit

Orlando, Florida, 32806, United States
Weill Cornell Medicine

New York, New York, 10021, United States

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