Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
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 for patients who are APOE4 homozygotes. It is currently in Phase 1 clinical development and has not been approved by the FDA. The therapy is designed to address the genetic risk associated with the APOE4 variant in this patient population.
LX1001 targets the APOE4 gene, which is a genetic risk factor for Alzheimer Disease. As a gene therapy, it aims to modify or compensate for the effects of the APOE4 variant. The drug is being studied in patients who carry two copies of the APOE4 gene, known as homozygotes.
LX1001 is being developed by Lexeo Therapeutics, Inc., a biopharmaceutical company. The company is conducting clinical trials to evaluate the safety and efficacy of this gene therapy for Alzheimer Disease in patients with the APOE4 genotype.
LX1001 is in Phase 1 clinical development. It is an investigational drug and has not been approved by regulatory authorities. The Phase 1 trials are designed to assess safety, tolerability, and preliminary efficacy in patients with Alzheimer Disease who are APOE4 homozygotes.
LX1001 has been studied in two clinical trials. The first, NCT03634007, was a completed Phase 1 study in patients with Alzheimer Disease and early onset Alzheimer Disease. The second, NCT05400330, is an active long-term follow-up study in Alzheimer Disease patients. Both trials are based in the United States.
LX1001 is a gene therapy specifically designed for patients with Alzheimer Disease who are APOE4 homozygotes. It targets the APOE4 gene, which is a known genetic risk factor for the disease. The therapy is being investigated to potentially address this genetic cause in the affected population.