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LX2020 · 1 trial · 3 indications
Evaluation of Safety and Tolerability of LX2020
| Arm | Type | Description |
|---|---|---|
| LX2020 | EXPERIMENTAL | Single ascending dose of LX2020, with a starting dose of 2.0 x10\^13 gc/kg, in multiple cohorts |
| Name | Type | Description |
|---|---|---|
| LX2020 | GENETIC | LX2020 is an adeno-associated viral vector encoding the human Plakophilin-2 (PKP2) gene (AAVrh.10hPKP2) |
Selected Inclusion Criteria: * Adults with a clinical diagnosis of ACM meeting the 2010 revised Task Force Criteria (TFC) * Genetic testing documenting a pathogenic or likely pathogenic variant in PKP2 * Frequent premature ventricular complexes (PVCs) * Implantable cardioverter-defibrillator (ICD) ...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Lexeo Therapeutics, Inc. | LXEO | 3 | PHASE1 | LX2020 |
LX2020 is an investigational gene therapy being developed for arrhythmogenic cardiomyopathy, a rare inherited heart condition. It is specifically intended for patients whose disease is caused by a pathogenic variant in the PKP2 gene. It is not approved for any use and remains in clinical development.
LX2020 targets PKP2, the gene encoding plakophilin-2. In PKP2-associated arrhythmogenic cardiomyopathy, loss of functional plakophilin-2 disrupts the heart's desmosomes, the structures that hold cardiac muscle cells together. LX2020 is designed as a gene therapy to address this underlying genetic cause.
LX2020 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 the sponsor of the clinical program evaluating LX2020 in patients with PKP2-associated arrhythmogenic cardiomyopathy.
LX2020 is in Phase 1 clinical development. It is an investigational therapy and has not been approved by the FDA. The FDA has granted LX2020 Fast Track, Orphan Drug, and Regenerative Medicine Advanced Therapy (RMAT) designations for its development in PKP2-associated arrhythmogenic cardiomyopathy.
LX2020 is being evaluated in the Phase 1 trial NCT06109181, titled Gene Therapy for ACM Due to a PKP2 Pathogenic Variant. The trial is active but not recruiting, is enrolling up to 10 participants aged 18 years and older in the United States, and is an uncontrolled, non-randomized study.