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Also known as Low dose LX2006, Mid Dose LX2006
LX2006 · 2 trials · 2 indications
| Arm | Type | Description |
|---|---|---|
| LX2006 | EXPERIMENTAL | Cohort 1: Participants ≥16 years of age with FA-CM Cohort 2: Participants ≥6 to \<16 years of age with FA-CM. As Cohort 2 will not be randomized, all participants will receive LX2006 upon enrollment. Participants in Cohort 2 will be enrolled after safety is assessed in a group of participants in Cohort 1. |
| Usual Care | OTHER | Cohort 1: Participants ≥16 years of age with FA-CM Participants will receive usual care for 26 weeks before receiving treatment with LX2006 (single crossover). |
| Cohort 1/ Cohort 2/ Cohort 3 | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| LX2006 | GENETIC | Adeno-associated viral vector encoding the FXN gene (AAVrh.10hFXN) |
| Usual Care | OTHER | Cohort 1: Participants ≥16 years of age with FA-CM Participants will receive usual care for 26 weeks before receiving treatment with LX2006 (single crossover). |
| Low dose LX2006 | GENETIC | Adeno-associated viral vector encoding the FXN gene (AAVrh.10hFXN) |
| Mid Dose LX2006 | GENETIC | Adeno-associated viral vector encoding the FXN gene (AAVrh.10hFXN) |
| High Dose LX2006 | GENETIC | Adeno-associated viral vector encoding the FXN gene (AAVrh.10hFXN) |
Inclusion Criteria: * Male or female, age at least 6 years at the time of signing the informed consent (and assent, if applicable). * Diagnosis of FA, based on clinical phenotype and genotype (GAA expansion on the frataxin gene) * Onset of FA on or before 25 years of age * Confirmed left ventricula...
LX2006 is an investigational gene therapy developed by Lexeo Therapeutics, Inc. (ticker LXEO) for Friedreich Ataxia. It is designed to deliver the FXN gene and is being studied in patients with Friedreich Ataxia and associated cardiomyopathy. LX2006 is in Phase 2 clinical development and has received multiple FDA designations, including Breakthrough Therapy, RMAT, Orphan Drug, Fast Track, and Rare Pediatric Disease.
LX2006 targets FXN, the gene that encodes frataxin. Friedreich Ataxia is associated with reduced frataxin levels, and LX2006 is a gene therapy intended to deliver the FXN gene. By targeting FXN, the therapy aims to address the underlying genetic deficiency in patients with Friedreich Ataxia and associated cardiomyopathy.
LX2006 is being developed by Lexeo Therapeutics, Inc., which trades under the ticker LXEO. Lexeo Therapeutics is the sponsor of the clinical trials evaluating LX2006 in Friedreich Ataxia. The program has received several FDA designations, including Breakthrough Therapy, RMAT, Orphan Drug, Fast Track, Rare Pediatric Disease, and Accelerated Approval.
LX2006 is in Phase 2 clinical development. It is an investigational gene therapy and has not been approved by the FDA. The Phase 2 study is currently recruiting, and an earlier Phase 1 trial has been completed with active follow-up. LX2006 has received FDA designations including Breakthrough Therapy, RMAT, Orphan Drug, Fast Track, Rare Pediatric Disease, and Accelerated Approval.
LX2006 is being evaluated in two clinical trials. NCT07721025 is a Phase 2 study of LX2006 gene therapy in Friedreich Ataxia cardiomyopathy that is currently recruiting in the United States, with an enrollment of 26 participants. NCT05445323 is a Phase 1 gene therapy trial for cardiomyopathy associated with Friedreich's Ataxia that is active but not recruiting, with 8 participants.
Yes, Low dose LX2006 and Mid Dose LX2006 are alternative names for LX2006. They refer to different dose levels of the same investigational gene therapy developed by Lexeo Therapeutics, Inc. (ticker LXEO) for Friedreich Ataxia. LX2006 is in Phase 2 clinical development and targets the FXN gene.