Lexeo Therapeutics
Clinical-stage biotechnology company based in New York City focused on developing gene and related therapies that address underlying genetic causes of cardiovascular and neurological diseases. The company advances AAV-based gene therapies, small molecules, protein replacement approaches and BBB-targeting delivery strategies through discovery, preclinical, and clinical development, and pursues regulatory designations and CMC readiness for registrational programs.
Industries
Nr. of Employees
medium (51-250)
Lexeo Therapeutics
Products
LX2006
AAV-based gene therapy candidate to deliver the FXN gene for treatment of Friedreich ataxia cardiomyopathy; being evaluated in Phase 1/2 and registrational trials and natural history studies.
LX2020
AAV-based gene therapy candidate designed to deliver a functional PKP2 gene to cardiac muscle for treatment of PKP2-associated arrhythmogenic cardiomyopathy; evaluated in an open-label dose-ascending Phase 1/2 trial.
LX2021
Gene therapy candidate to deliver a functional GJA1 (connexin 43) gene to cardiac muscle to address desmoplakin-associated cardiomyopathy and related arrhythmogenic cardiomyopathies.
LX2022
Gene therapy candidate to deliver a functional TNNI3 gene to myocardial cells for treatment of a TNNI3-associated form of hypertrophic cardiomyopathy.
LX1001 / LX1020 / LX1021
Series of CNS-directed gene therapy candidates designed to express protective APOE2 variants and/or suppress APOE4 in the CNS for treatment of APOE4 homozygous Alzheimer's disease patients.
LX3010 and preclinical small-molecule candidates
Oral combination therapy (LX3010) and discovery-stage small molecules intended to increase frataxin expression and address mitochondrial dysfunction and oxidative stress in Friedreich ataxia.
LX2006
AAV-based gene therapy candidate to deliver the FXN gene for treatment of Friedreich ataxia cardiomyopathy; being evaluated in Phase 1/2 and registrational trials and natural history studies.
LX2020
AAV-based gene therapy candidate designed to deliver a functional PKP2 gene to cardiac muscle for treatment of PKP2-associated arrhythmogenic cardiomyopathy; evaluated in an open-label dose-ascending Phase 1/2 trial.
LX2021
Gene therapy candidate to deliver a functional GJA1 (connexin 43) gene to cardiac muscle to address desmoplakin-associated cardiomyopathy and related arrhythmogenic cardiomyopathies.
LX2022
Gene therapy candidate to deliver a functional TNNI3 gene to myocardial cells for treatment of a TNNI3-associated form of hypertrophic cardiomyopathy.
LX1001 / LX1020 / LX1021
Series of CNS-directed gene therapy candidates designed to express protective APOE2 variants and/or suppress APOE4 in the CNS for treatment of APOE4 homozygous Alzheimer's disease patients.
LX3010 and preclinical small-molecule candidates
Oral combination therapy (LX3010) and discovery-stage small molecules intended to increase frataxin expression and address mitochondrial dysfunction and oxidative stress in Friedreich ataxia.
Expertise Areas
- Gene therapy development
- AAV vector manufacturing and CMC
- Preclinical model development and biodistribution
- Clinical trial management for rare and genetic diseases
Key Technologies
- AAV vector-mediated gene delivery
- Baculovirus/Sf9 suspension manufacturing
- HEK293 adherent production processes
- iPSC-derived cardiomyocyte in vitro platforms
News & Updates
JAMA Cardiology publication reporting on AAV-based gene therapy for Friedreich ataxia cardiomyopathy.
Publication describing preclinical results for APOE2-Christchurch variant delivery to suppress pathology in AD models.
Program awarded Breakthrough Therapy, RMAT, Rare Pediatric Disease, Orphan Drug, Fast Track designations and participation in FDA CMC Development and Readiness Pilot.
Program awarded Orphan Drug, Fast Track and RMAT designations by the FDA.
Multiple publications and presentations across journals and conferences (JAMA Cardiology, ASGCT, ACC) describing preclinical and clinical data for cardiac and FA programs.
JAMA Cardiology publication reporting on AAV-based gene therapy for Friedreich ataxia cardiomyopathy.
Publication describing preclinical results for APOE2-Christchurch variant delivery to suppress pathology in AD models.
Program awarded Breakthrough Therapy, RMAT, Rare Pediatric Disease, Orphan Drug, Fast Track designations and participation in FDA CMC Development and Readiness Pilot.
Program awarded Orphan Drug, Fast Track and RMAT designations by the FDA.
Multiple publications and presentations across journals and conferences (JAMA Cardiology, ASGCT, ACC) describing preclinical and clinical data for cardiac and FA programs.