Long-Term Follow-up Study of ADVM-043

ADVM-043-03 is a long-term follow-up safety study of subjects in the ADVM 043 01 gene therapy
clinical study (ADVANCE) delivering ADVM-043 for treatment of Alpha-1 Antitrypsin (A1AT)
deficiency. Subjects will roll over after 12 months in the ADVANCE study into this study for
continued safety monitoring. ADVM-043-03 is a multi-site observational study, without
administration of investigational product, evaluating the long-term safety of ADVM-043
administered in a prior study. Subjects who received ADVM-043 will be followed for up to 3
years post-treatment

Key Inclusion Criteria:

- The subject has A1AT deficiency and has previously received ADVM-043 gene therapy

Key Exclusion Criteria:

- The subject is unwilling or unable to participate in all required study evaluations in
the long-term follow-up protocol

- The subject is participating in another investigational treatment protocol

- The subject is unable to understand the purpose and risks of the study or cannot
provide a signed and dated informed consent form (ICF)