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Also known as AAVrh.10hAPOE2
LX1001 · 2 trials · 2 indications
All emergent adverse events will be collected
All incidents of serious adverse events will be collected
Adverse events categorized and graded
Adverse events categorized and graded per study drug dose
| Arm | Type | Description |
|---|---|---|
| Previously administered LX1001 | EXPERIMENTAL | This is a long-term follow-up study to evaluate the safety following LX1001, a gene therapy, for participants who are APOE4 homozygotes with clinical diagnoses varying from MCI or dementia due to AD who have previously received LX1001. Study LX1001-01 was designed to assess the safety of LX1001 at 4 ascending doses (1.4 × 1010, 4.4 × 1010, 1.4 × 1011 gene copy \[gc\]/mL CSF and 1.4 x 1014 \[fixed dose\]) as per droplet digital polymerase chain reaction methodology, with each group consisting of approximately n=3-5 individuals for a total of approximately 15 participants for the entire study. In this study, participants who have received LX1001 in the parent protocol (LX1001-01) will be followed for up to 260 weeks post gene therapy administration |
| Cohort 1: 1.4 x 10^10 gc/mL CSF | EXPERIMENTAL | Participants will receive 1.4 x 10\^10 gc/mL CSF of LX1001. |
| Cohort 2: 4.4 x 10^10 gc/mL CSF | EXPERIMENTAL | Participants will receive 4.4 x 10\^10 gc/mL CSF of LX1001. |
| Cohort 3: 1.4 x 10^11 gc/mL CSF | EXPERIMENTAL | Participants will receive 1.4 x 10\^11 gc/mL CSF of LX1001. |
| Cohort 4: 1.4 x 10^14 gc (fixed dose) | EXPERIMENTAL | Participants will receive 1.4 x 10\^14 gc (fixed dose; approximately 3.4 × 10\^11 gc/mL CSF based on an average CSF volume of 409 mL) of LX1001. |
| Name | Type | Description |
|---|---|---|
| LX1001 | BIOLOGICAL | Gene therapy |
Inclusion Criteria: * Participants who received LX1001 in study LX1001-01 Exclusion Criteria: * Participants with any clinically significant medical condition that, in the opinion of the investigator, would pose a risk to participant safety * Participants who agree not to post their personal medi...
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LX1001 is an investigational gene therapy being developed for Alzheimer disease, specifically in patients who are homozygous for the APOE4 allele. It is designed to deliver the APOE2 gene to the central nervous system. It is not approved for any indication and remains in clinical development.
LX1001 targets APOE4, the genetic variant that is the strongest risk factor for late-onset Alzheimer disease. The therapy delivers the APOE2 gene to the central nervous system, aiming to provide the protective APOE2 protein in APOE4 homozygotes. It is a gene therapy, not a monoclonal antibody.
LX1001 is being developed by Lexeo Therapeutics, Inc., a clinical-stage gene therapy company. Lexeo Therapeutics trades on the Nasdaq under the ticker symbol LXEO. The company is focused on developing gene therapies for cardiovascular and neurological diseases, including Alzheimer disease.
LX1001 is in Phase 1 clinical development. It is an investigational gene therapy and has not been approved by the FDA or any regulatory authority. Two Phase 1 trials have been conducted, one completed and one active but not recruiting, with a total enrollment of 25 patients.
LX1001 has been studied in two Phase 1 trials. NCT03634007, titled Gene Therapy for APOE4 Homozygote of Alzheimer's Disease, is completed with 15 patients. NCT05400330, a long-term follow-up study, is active but not recruiting with 10 patients. Both trials enrolled patients aged 50 and older in the United States.
No, LX1001 is a gene therapy, not a monoclonal antibody. It uses a viral vector to deliver the APOE2 gene to the central nervous system. The therapy is being studied in APOE4 homozygote Alzheimer disease patients, a biomarker-selected population.