Summary
Long-term follow-up of subjects who received SB-318, SB-913, or SB-FIX in a previous trial and completed at least 52 weeks post-infusion follow-up in their primary protocol. Enrolled subjects will be followed for a total of up to 10 years following exposure to SB-318, SB-913, or SB-FIX.
Official Title
Long-Term Follow-up of Subjects Who Were Treated With SB-318, SB-913, or SB-FIX, for Targeted Genome Editing Into the Albumin Gene in the Liver
Details
Non-interventional, multi-center, long-term follow-up (LTFU) study of subjects dosed with SB-318 in the clinical study SB-318-1502, SB-913 in the clinical study SB-913-1602, and SB-FIX in clinical study SB-FIX-1501. All subjects dosed in the studies and completed at least 52 weeks post-infusion follow-up in their primary protocol will be offered to participate. Subjects who enroll will be monitored for a total of up to 10 years following exposure to the respective investigational products.
Keywords
Hemophilia B, Mucopolysaccharidosis I, Mucopolysaccharidosis II, Hurler-Scheie Syndrome, Hunter Syndrome, Gene Editing, Gene Therapy, Zinc Finger, SB-318, SB-913, SB-FIX, Rare, Genetic, DNA, Sangamo, ZFN, Long Term, MPS, Hemophilia A, Mucopolysaccharidoses
