Cardiac
programs

Advancing Gene Therapy in Monogenic Diseases

Despite recent medical advances in the rare cardiovascular field, many rare cardiac diseases remain underdiagnosed, undertreated, or untreated. Therefore, there is an urgent need for new treatments to address these conditions for patients. We are developing a number of disease-modifying gene therapy candidates to treat larger-rare cardiovascular diseases that have significant unmet need and no approved disease-modifying treatments. Our most advanced program, LX2006, is an AAV-based gene therapy candidate for the treatment of FA cardiomyopathy caused by mutations in the FXN gene. We are also advancing several other AAV-based gene therapy programs to treat additional genetically defined cardiac diseases, including LX2020 to treat ARVC caused by mutations in the PKP2 gene, LX2021 to treat ARVC associated with Cx43 deficiency, and LX2022 to treat HCM associated with mutations in the TNNI3 gene.

LIST OF PROGRAMS

LX2006

Cardiac Friedreich’s Ataxia

Friedreich’s ataxia (FA) is a rare degenerative multi-system disorder affecting about one in 40,000 people in the United States. FA is caused by a gene mutation that disrupts the normal production of an important protein called frataxin, that functions in the mitochondria (the energy producing factories) of the cell. Symptoms generally begin in childhood. Patients develop progressive muscle incoordination and weakness, and most develop heart disease, typically hypertrophic cardiomyopathy, as well as arrythmias. FA often leads to shortened lifespan. The majority of FA related deaths are due to heart failure.

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LX2021

Arrhythmogenic cardiomyopathy (CX43)

Arrhythmogenic right ventricular cardiomyopathy is a genetic heart disease frequently caused by genetic mutations that impair the structure of desmosomes, which connect heart muscle cells, resulting in cardiac cell death, fibrosis, heart dysfunction, rhythm abnormalities, and sudden death. The US prevalence of ARVC is estimated at 1:2000-1:5000 people. More than 40% of ARVC patients die or have heart transplantation within 10 years of diagnosis.

LX2020

Arrhythmogenic cardiomyopathy (PKP2)

Arrhythmogenic right ventricular cardiomyopathy is a genetic heart disease frequently caused by genetic mutations that impair the structure of desmosomes, which connect heart muscle cells, resulting in cardiac cell death, fibrosis, heart dysfunction, rhythm abnormalities, and sudden death. The US prevalence of ARVC is estimated at 1:2000-1:5000 people. More than 40% of ARVC patients die or have heart transplantation within 10 years of diagnosis.

LX2022

Hypertrophic cardiomyopathy (TNNI3)

Hypertrophic cardiomyopathy (HCM) is one of the most common forms of genetic cardiomyopathy and is caused by mutations that affect the cardiac sarcomere in approximately 75% of cases. It is inherited as an autosomal dominant trait, with over 500,000 patients in the United States alone who have a genetic form of HCM.