Gene therapy targets Alzheimer's in High-Risk group
NCT ID NCT03634007
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.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Duke University
Durham, North Carolina, 27708, United States
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K2 Medical Research
Maitland, Florida, 32751, United States
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PPD- Orlando Research Unit
Orlando, Florida, 32806, United States
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Weill Cornell Medicine
New York, New York, 10021, United States