Drugs / LX2020
last change Aug 2026 re-read 3 minutes ago

LX2020

also known as AAVrh.10hPKP2

Gene therapy (AAV / viral vector) targets PKP2

Developed for
arrhythmogenic right ventricular dysplasia 9
Investigated by
Lexeo Therapeutics

Trials 1

202520262027
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 1/2 NCT06109181 Feb 2024 → Dec 2026 expected arrhythmogenic right ventricular dysplasia 9 Lexeo Therapeutics Active not recruiting No outcome recorded

News releases announcing trial results or a regulatory action · 6

DateIssuerRelease
2026-08-05 Lexeo Therapeutics Regulatory Lexeo Therapeutics Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for LX2020 for the Treatment of PKP2 Arrhythmogenic Cardiomyopathy lexeotx.com ↗
2026-01-12 Lexeo Therapeutics Results Lexeo Therapeutics Announces Positive Interim Phase I/II Data for LX2020 for the Treatment of PKP2-Associated Arrhythmogenic Cardiomyopathy lexeotx.com ↗
2025-03-24 Lexeo Therapeutics Results Lexeo Therapeutics Reports Fourth Quarter and Full Year 2024 Financial Results and Operational Highlights lexeotx.com ↗
Interim update from cohort 1 of LX2020 HEROIC-PKP2 Phase 1/2 trial: observed 71% and 115% increases in PKP2 protein expression in first two post-treatment biopsies; first participant evaluated 6-months after dosing experienced 67% reduction in premature ventricular contractions (PVCs)
2023-12-18 Lexeo Therapeutics Regulatory Lexeo Therapeutics Granted FDA Fast Track Designation and Orphan Drug Designation for LX2020, an AAV-Based Gene Therapy Candidate for PKP2 Arrhythmogenic Cardiomyopathy (ACM) lexeotx.com ↗
today announced the U.S. Food and Drug Administration (FDA) has granted Fast Track designation and Orphan Drug designation to LX2020
2023-08-01 Lexeo Therapeutics Regulatory LEXEO Therapeutics Announces FDA Clearance of IND for LX2020, an AAV-based Gene Therapy Candidate for PKP2 Arrhythmogenic Cardiomyopathy lexeotx.com ↗
LEXEO Therapeutics (LEXEO), a clinical-stage gene therapy company advancing adeno-associated virus (AAV)-based gene therapy candidates for genetically defined cardiovascular diseases and a genetically defined sub-group of Alzheimer’s disease, today announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for LX2020.
2023-05-02 Lexeo Therapeutics Results LEXEO Therapeutics Announces Data Presentations at the 26th American Society of Gene & Cell Therapy (ASGCT) Annual Meeting lexeotx.com ↗
These data indicate that LX2020 overcomes cardiac PKP2 deficits in ARVC settings by driving human PKP2 expression and that this is sufficient to prevent and stabilize the progression of rapid ARVC disease in a novel PKP2 genetic mouse model.

Evidence & citations 5 cited values

Every value below carries the sentence it was read from. 1 source stands behind the page.

FieldValueCited text
Known as LX2020 ClinicalTrials.gov intervention name — accepted as the source's own label NCT06109181 ↗
Known as AAVrh.10hPKP2 “LX2020 is an adeno-associated viral vector encoding the human Plakophilin-2 (PKP2) gene (AAVrh.10hPKP2)” NCT06109181 ↗
Modality Gene therapy (AAV / viral vector) “adeno-associated viral vector” NCT06109181 ↗
Route Intravenous “intravenous” NCT06109181 ↗
Target PKP2 “encoding the human Plakophilin-2 (PKP2) gene” NCT06109181 ↗