Lexeo Therapeutics
Lexeo Therapeutics works on 2 rare diseases tracked on Trial Friend, including Arrhythmogenic Cardiomyopathy, Friedreich Ataxia, with 5 active clinical trials (3 recruiting).
Lexeo Therapeutics is a New York gene therapy company focused on genetic heart disease, a space most gene therapy developers avoided while chasing neurological and blood disorders. Its lead program, LX2006, delivers a working copy of the frataxin gene to heart muscle in Friedreich ataxia, where cardiomyopathy, not the better-known neurological decline, is the leading cause of death.
The program has collected nearly every FDA acceleration mechanism available: Breakthrough Therapy, Regenerative Medicine Advanced Therapy, Fast Track, and orphan drug designations, on the strength of interim Phase 1/2 data showing reductions in abnormal left ventricular mass and improvements in cardiac biomarkers. In 2026 Lexeo launched SUNRISE-FA 2, the pivotal study intended to support an accelerated-approval BLA in 2028. For Friedreich ataxia families, it is the first serious attempt to treat the disease's deadliest feature at its genetic root.
The second program, LX2020, targets PKP2 arrhythmogenic cardiomyopathy, an inherited arrhythmia disease that can cause sudden cardiac death in young, otherwise healthy people, and holds its own RMAT designation. Both programs use AAV delivery refined for cardiac tissue, the company's core bet.
Lexeo Therapeutics Drug Pipeline
Lexeo Therapeutics has 5 active clinical trials across 2 development stages, with 3 currently recruiting participants. Clinical trials advance through phases: Phase 1 tests safety in a small group, Phase 2 evaluates effectiveness and side effects, Phase 3 confirms benefit in a larger population, and Phase 4 monitors long-term safety after FDA approval.
Note: This pipeline counts the active and recruiting trials listed on this page, from ClinicalTrials.gov. For companies that work outside rare disease, we list only trials for rare diseases we track, so the company's full pipeline may be larger.
Lexeo Therapeutics Clinical Trials (5)
Active and recruiting clinical trials sponsored by Lexeo Therapeutics, sourced live from ClinicalTrials.gov. Each trial card shows the study phase, current recruitment status, conditions under investigation, study locations, eligibility criteria, and a direct link to the full ClinicalTrials.gov record. You can also download a one-page PDF summary to share with your doctor.
Note: Recruitment statuses on ClinicalTrials.gov may not immediately reflect recent FDA decisions, sponsor announcements, or enrollment changes. Always confirm a trial's current status directly with the study coordinator.
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FDA-Approved Drugs
Lexeo Therapeutics Trial Locations
Lexeo Therapeutics clinical trials are running at 34 sites in 8 countries. Click any country to drill down by state, city, and individual research facility. Proximity to a trial site is one of the most important factors in deciding whether to participate.
Rare Disease Focus Areas (2)
Diseases targeted by Lexeo Therapeutics's clinical trial and drug development programs
Arrhythmogenic cardiomyopathy is a rare genetic heart disease characterized by replacement of cardiac myocardium with fibrofatty tissue, particularly affecting the right ventricle. Caused by mutations...
Friedreich ataxia is an autosomal recessive neurodegenerative disease causing progressive damage to the nervous system, resulting in loss of coordination (ataxia), weakness, and heart problems. The co...
Patient Resources
Organizations and resources related to Lexeo Therapeutics's rare disease focus areas
Frequently Asked Questions About Lexeo Therapeutics
Common questions about Lexeo Therapeutics's rare disease programs, clinical trials, and treatments.