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LX2020

Phase 1

Arrhythmogenic Cardiomyopathy | Gene therapy | Rare Disease |Lexeo Therapeutics, Inc.|Last Updated: Nov 12, 2025

Target and mechanism

Molecular targetPKP2
ModalityGene therapy

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLEDDMC
Total Trials1
Total Enrollment10

FDA Designations

FAST_TRACKORPHAN_DRUG

Clinical trial landscape

LX2020 · 1 trial · 3 indications

Phase 1 1
NCT06109181Gene Therapy for ACM Due to a PKP2 Pathogenic VariantArrhythmogenic Cardiomyopathy
ACTIVE NOT_RECRUITING10 Analytics
PHASE1ACTIVE NOT_RECRUITING
Gene Therapy for ACM Due to a PKP2 Pathogenic Variant
Arrhythmogenic CardiomyopathyUnlock trial analytics

Study Endpoints

Primary Endpoints

Percentage of subjects who experienced at least 1 treatment emergent adverse event (TEAE) and/or 1 treatment emergent serious adverse event (TESAE).
12 months

Evaluation of Safety and Tolerability of LX2020

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Study Design & Arms

AllocationNA
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
LX2020EXPERIMENTALSingle ascending dose of LX2020, with a starting dose of 2.0 x10\^13 gc/kg, in multiple cohorts

Interventions

NameTypeDescription
LX2020GENETICLX2020 is an adeno-associated viral vector encoding the human Plakophilin-2 (PKP2) gene (AAVrh.10hPKP2)
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Eligibility Criteria

Age Range18 Years to 65 Years
SexALL
Healthy VolunteersNo
Study Sites5

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) ...

Countries:United States
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Competitive Landscape -Arrhythmogenic Right Ventricular Cardiomyopathy 3 trials (matched to "Arrhythmogenic Cardiomyopathy")

CompanyTickerTrialsLead PhaseDrugs
Lexeo Therapeutics, Inc.LXEO3PHASE1LX2020
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Recent Changes (Last 90 Days)

LOWMay 26, 2026NCT06109181primaryCompletionDate: changed
LOWMay 24, 2026NCT06109181studyFirstPostDate: changed

Frequently asked questions about LX2020

What is LX2020 used for?

LX2020 is an investigational gene therapy being developed for arrhythmogenic cardiomyopathy, a rare heart condition. It is designed to treat patients with a PKP2 pathogenic variant, which is a genetic cause of the disease. The therapy is currently in Phase 1 clinical development.

What does LX2020 target?

LX2020 targets the PKP2 gene. It is a gene therapy intended to address arrhythmogenic cardiomyopathy caused by a PKP2 pathogenic variant. By targeting this genetic cause, the therapy aims to treat the underlying condition.

Who makes LX2020?

LX2020 is being developed by Lexeo Therapeutics, Inc., a biopharmaceutical company. Lexeo Therapeutics is publicly traded under the ticker symbol LXEO on the stock market.

What phase is LX2020 in?

LX2020 is in Phase 1 clinical development. It is an investigational drug, meaning it has not been approved by regulatory authorities and is still being studied in clinical trials to evaluate its safety and efficacy.

What clinical trials is LX2020 in?

LX2020 is being studied in a Phase 1 clinical trial with the identifier NCT06109181. This trial is titled 'Gene Therapy for ACM Due to a PKP2 Pathogenic Variant' and is currently active but not recruiting participants. The trial is enrolling 10 participants in the United States.

Is LX2020 the same as PKP2-ACM?

LX2020 is not the same as PKP2-ACM. PKP2-ACM is a condition, specifically arrhythmogenic cardiomyopathy caused by a PKP2 pathogenic variant. LX2020 is an investigational gene therapy being developed to treat this condition.