Lexeo Therapeutics
Lexeo Therapeutics is a clinical-stage genetic medicine company based in New York City, dedicated to transforming healthcare by developing gene therapies targeting genetic cardiovascular and Alzheimer’s diseases. Their mission is to create meaningful genetic medicines for diseases regardless of prevalence, aiming to resolve the burden of disease and improve societal health. They leverage pioneering science, strategic partnerships, and a stepwise development approach to advance their pipeline of AAV-based gene therapy candidates. The company is committed to patient engagement, diversity, and ethical principles in clinical development.
Industries
Nr. of Employees
small (1-50)
Lexeo Therapeutics
Products
AAV gene therapy candidate delivering frataxin (FXN) for Friedreich’s ataxia cardiomyopathy
A CNS-excluded cardiac-directed AAV candidate designed to restore frataxin expression in myocardial cells to treat Friedreich’s ataxia–associated cardiomyopathy; evaluated in an open-label Phase 1/2 clinical trial (SUNRISE-FA).
AAV gene therapy candidate delivering PKP2 for PKP2-associated arrhythmogenic cardiomyopathy
Vector designed to deliver a functional PKP2 gene to cardiac muscle to increase desmosomal protein levels and restore myocardial cell function for PKP2-ACM.
AAV gene therapy candidate delivering connexin 43 (Cx43) for Desmoplakin-related cardiomyopathy
Therapeutic approach to express Cx43 in myocardium to address loss of connexin 43 observed across certain forms of arrhythmogenic and dilated cardiomyopathy, initially targeting DSP cardiomyopathy.
AAV gene therapy candidate delivering TNNI3 for hypertrophic cardiomyopathy
Vector designed to deliver a functional TNNI3 gene to myocardial cells to treat forms of hypertrophic cardiomyopathy caused by TNNI3 mutations.
CNS AAV gene therapy candidate expressing APOE2 for APOE4‑associated Alzheimer’s disease
CNS-directed AAV candidate intended to express the APOE2 allele in APOE4 homozygous patients to slow or halt Alzheimer’s disease progression; under evaluation in a Phase 1/2 dose‑escalation trial (LEAD).
CNS AAV gene therapy candidate delivering Christchurch‑variant APOE2
Gene therapy approach delivering an APOE2 allele modified with the Christchurch mutation to enhance neuroprotective effects and reduce pathological spread in Alzheimer’s disease models.
AAV gene therapy candidate delivering frataxin (FXN) for Friedreich’s ataxia cardiomyopathy
A CNS-excluded cardiac-directed AAV candidate designed to restore frataxin expression in myocardial cells to treat Friedreich’s ataxia–associated cardiomyopathy; evaluated in an open-label Phase 1/2 clinical trial (SUNRISE-FA).
AAV gene therapy candidate delivering PKP2 for PKP2-associated arrhythmogenic cardiomyopathy
Vector designed to deliver a functional PKP2 gene to cardiac muscle to increase desmosomal protein levels and restore myocardial cell function for PKP2-ACM.
AAV gene therapy candidate delivering connexin 43 (Cx43) for Desmoplakin-related cardiomyopathy
Therapeutic approach to express Cx43 in myocardium to address loss of connexin 43 observed across certain forms of arrhythmogenic and dilated cardiomyopathy, initially targeting DSP cardiomyopathy.
AAV gene therapy candidate delivering TNNI3 for hypertrophic cardiomyopathy
Vector designed to deliver a functional TNNI3 gene to myocardial cells to treat forms of hypertrophic cardiomyopathy caused by TNNI3 mutations.
CNS AAV gene therapy candidate expressing APOE2 for APOE4‑associated Alzheimer’s disease
CNS-directed AAV candidate intended to express the APOE2 allele in APOE4 homozygous patients to slow or halt Alzheimer’s disease progression; under evaluation in a Phase 1/2 dose‑escalation trial (LEAD).
CNS AAV gene therapy candidate delivering Christchurch‑variant APOE2
Gene therapy approach delivering an APOE2 allele modified with the Christchurch mutation to enhance neuroprotective effects and reduce pathological spread in Alzheimer’s disease models.
Services
Early‑phase clinical development and trial execution
Design and execution of Phase 1/2 clinical trials for investigational gene therapies, including site operations, vendor oversight, and regulatory-compliant trial documentation.
Preclinical discovery and in vivo pharmacology
In vitro screening, cellular assays, RNA-seq analysis, and in vivo efficacy testing in rodent disease models to support candidate selection and translational strategy.
AAV CMC development and manufacturing scale-up
Process development and scale-up for AAV manufacturing on insect cell/baculovirus platforms and analytical support for product quality attributes.
Early‑phase clinical development and trial execution
Design and execution of Phase 1/2 clinical trials for investigational gene therapies, including site operations, vendor oversight, and regulatory-compliant trial documentation.
Preclinical discovery and in vivo pharmacology
In vitro screening, cellular assays, RNA-seq analysis, and in vivo efficacy testing in rodent disease models to support candidate selection and translational strategy.
AAV CMC development and manufacturing scale-up
Process development and scale-up for AAV manufacturing on insect cell/baculovirus platforms and analytical support for product quality attributes.
Expertise Areas
- AAV gene therapy development
- Genetic cardiovascular disease therapies
- APOE4-associated Alzheimer’s disease therapeutics
- CMC and bioprocess scale-up for viral vectors
Key Technologies
- Adeno-associated virus (AAV) gene delivery
- Sf9-baculovirus production platform for AAV
- miRNA-mediated gene suppression
- RNA-seq and transcriptomic analysis