Topline results from Phase 1/2 AAV gene therapy (LX1001) in APOE4/4 homozygotes with Alzheimer’s disease
LX1001 is the first investigational gene therapy for APOE4 homozygotes. Data suggest LX1001 is generally safe and well tolerated with reduction in CSF tau biomarkers and Tau PET, measures that are highly correlated with cognitive decline.
Authors
- R. G. Crystal
- Sandi See Tai
- Michael G. Kaplitt (ORCID: https://orcid.org/0000-0002-8979-2459)
- Kim G. Johnson (ORCID: https://orcid.org/0000-0002-8793-2489)
- Dolan Sondhi (ORCID: https://orcid.org/0000-0002-2218-5910)
- Richie Khanna
- Rosalind McLaine
- Gianni Amato
- Stephen Kaminsky
- Nithya Selvan
Institutions
- Duke University (US)
- Cornell University (US)
- Lexicon Pharmaceuticals (United States) (US)
Publication Details
- Journal
- Alzheimer s & Dementia
- Published
- 2025-12-01
- DOI
- https://doi.org/10.1002/alz70859_101538
- Primary Topic
- Virus-based gene therapy research
- Type
- article
- Field-Weighted Citation Impact
- 0.00