1. Ability to understand the purpose and risks of the study, as well as provide signed
and dated informed consent and authorization to use protected health information (PHI)
in accordance with national and local study participant privacy regulations.
2. Subjects who have received a single administration of SPK-9001, and completed the
required assessments in the SPK-9001-101 clinical study.
1. Subjects who will not consent for up to 5 years of safety follow-up.
2. Subjects who are unable or unwilling to comply with the study visits and requirements.
3. Subjects who have planned to participate in another interventional investigational
gene transfer study.
4. Subjects with any clinically significant medical condition that the Investigator
believes would pose as a safety risk or are not capable of the performing endpoints of
this study.