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

Inclusion Criteria:

1. Male or female ≥ 18 to ≤65 years of age

2. Pathogenic GLA mutation consistent with Fabry Disease

3. Confirmed diagnosis of classic or late-onset Fabry disease with cardiac involvement

4. Intolerant of ERT, unable or unwilling to receive ERT, or progressive diseasedespite ERT or migalastat

5. Individuals receiving ERT, must be on a stable dose for at least 6 months

6. Agree to use highly effective contraception

Exclusion Criteria:

1. Presence of pre-existing antibodies to 4D-310 capsid or to AGA

2. eGFR <65 mL/min/1.73 m2

3. Undergone kidney transplantation or currently on hemodialysis or peritoneal dialysis

4. HIV, active or chronic hepatitis B or C,

5. Evidence of liver disease, severe pulmonary disease or diabetes with poor glycemiccontrol

6. History of stroke or transient ischemic attack within the last 12 months, or othersignificant thromboembolic disease history (e.g. pulmonary embolism)

7. Contraindication to systemic corticosteroid therapy or immunosuppressive therapy

8. Chronic steroid use, defined as ≥ 3 months of oral corticosteroid use within thelast 12 months.

9. Moderately severe to severe cardiovascular disease or uncontrolled hypertension

10. Left ventricular ejection fraction of <45% on echocardiogram (ECHO)

11. Currently receiving investigational drug, device or therapy or having ever receivedgene therapy

12. History of infusion related response to ERT or any adverse reaction leading to ERTdiscontinuation

13. History of cancer within 2 years (exceptions include non-melanoma skin cancer,localized prostate cancer treated with curative intent)

14. Pregnant or breast-feeding

Note: Other inclusion and exclusion criteria apply