4D-310 in Adults With Fabry Disease and Cardiac Involvement

Inclusion Criteria: Adult males and females Pathogenic GLA mutation consistent with Fabry Disease Confirmed diagnosis of classic or late-onset Fabry disease with cardiac involvement Individuals on ERT must be on a stable dose for at least 6 months (and a minimum of 12 months total exposure) prior to study enrollment Agree to use highly effective contraception Exclusion Criteria: Presence of high titer neutralizing antibody to 4D-310 capsid, or presence of high antibody titer to AGA eGFR <45 mL/min/1.73 m2 Undergone kidney transplantation or currently on hemodialysis or peritoneal dialysis HIV, active or chronic hepatitis B or C, Evidence of liver disease, severe pulmonary disease or diabetes with poor glycemic control History of stroke or transient ischemic attack within the last 12 months, or other significant thromboembolic disease history (e.g. pulmonary embolism) Contraindication to systemic corticosteroid therapy or immunosuppressive therapy Chronic steroid use, defined as ≥ 3 months of oral corticosteroid use within the last 12 months. Moderately severe to severe cardiovascular disease or uncontrolled hypertension Left ventricular ejection fraction of <45% on echocardiogram (ECHO) Currently receiving investigational drug, device or therapy or having ever received gene therapy History of infusion related response to ERT or any adverse reaction leading to ERT discontinuation History of cancer within 2 years (exceptions include non-melanoma skin cancer, localized prostate cancer treated with curative intent) Pregnant or breast-feeding