A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subjects With BAG3 Dilated Cardiomyopathy

Prior treatment

• Previous participation in a study of gene transfer or gene editing.
• I.V. inotropic, vasodilator, or diuretic therapy ≤ 30 days prior to enrollment.
• Severe RV dysfunction assessed by echocardiogram or CMR ≤ 12 months prior to screening

Other illness

• CV disease that may be related to a genetic etiology other than a BAG3 pathogenic or likely pathogenic variant.
• History of intracardiac thrombosis or arterial thromboembolic events

Lab values

• LVEF \< 25% by echocardiogram or CMR at ≤ 3 months prior to screening

Other

• NYHA Class I or IV HF

• Subjects are eligible for inclusion into the study only if all the following criteria apply:
• Male or female between 18 and 65 years of age at the time of signing the informed consent
• Capable of and willing to provide signed informed consent
• Clinical diagnosis of DCM defined as and requiring each of the following:
  • Mild to moderate systolic dysfunction (LVEF ≥ 25% and ≤ 45%) by echocardiography or CMR performed within 3 months of enrollment.
  • Absence of severe coronary artery disease (\>70% stenosis) or active myocardial ischemia as the etiology of LV systolic dysfunction
  • Absence of uncontrolled hypertension, significant cardiac valve disease (i.e., greater than moderate in severity), infiltrative disorder, or systemic disease known to cause cardiomyopathy.
• Documentation of a pathogenic or likely pathogenic variant in BAG3
• History of ICD implantation ≥ 3 months prior to enrollment
• NYHA Class II or III HF symptoms with stable HF therapeutic guideline-directed medical regimen for 30 days prior to enrollment