Cardiac
programs
Advancing novel therapies in cardiovascular
genetic diseases.
Despite recent medical advances in the precision cardiovascular field, cardiovascular disease remains the most significant cause of morbidity and mortality in the western world and is rapidly becoming a primary cause of death worldwide. Therefore, there is an urgent need for new treatments to address these conditions for patients.
We are developing a number of disease-modifying therapeutic candidates to treat rare cardiovascular diseases that have significant unmet need and no approved treatments that address the underlying genetic cause of the disease.
CARDIOVASCULAR PROGRAMS
LX2020 is a gene therapy candidate designed to deliver a functional PKP2 gene to cardiac muscle for the treatment of PKP2 arrhythmogenic cardiomyopathy, or PKP2-ACM. PKP2 mutations are associated with approximately 75% of all genetic cases of ACM, and are estimated to affect approximately 60,000 individuals in the United States. PKP2 mutations can cause replacement of heart muscle with fibrotic and fatty tissue and are associated with severe arrhythmias, cardiac dysfunction, and sudden cardiac death.
LX2020 is designed to increase desmosomal PKP2 protein levels, reassemble desmosomes, and restore myocardial cell function. LX2020 is currently being evaluated in an open-label, dose-ascending Phase I/II clinical trial (HEROIC-PKP2).
The FDA has granted Orphan Drug, Fast Track, and RMAT designations to LX2020 for the treatment of PKP2-ACM.
Learn moreLX2021 is a gene therapy candidate we are developing to deliver a functional copy of the gene encoding connexin 43 (Cx43), a critical gap junction protein in the heart. It is being developed to treat a group of inherited cardiac muscle disorders associated with a high risk of sudden cardiac death. We believe restoring the Cx43 protein can potentially treat multiple genetic causes of arrhythmogenic cardiomyopathy (ACM) because the cardiac loss of Cx43 is a molecular deficit generally observed in all ACM patient populations.
Our LX2021 program is initially targeting desmoplakin, or DSP, cardiomyopathy, a distinct form of ACM as well as a certain form of dilated cardiomyopathy, impacting up to approximately 35,000 individuals in the United States.
Learn moreLX2022 is a gene therapy candidate we are developing to deliver a functional TNNI3 gene to myocardial cells to treat a distinct form of hypertrophic cardiomyopathy (HCM) due to mutations in the TNNI3 gene. TNNI3 is a critical component of the thin filament of the sarcomere. Mutations in the TNNI3 gene often result in left ventricular hypertrophy and restrictive cardiomyopathy, leading to arrhythmias and heart failure.
TNNI3 variants compose 3-5% of all HCM cases, impacting approximately 25,000 individuals in the United States.
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