The study will evaluate safety and efficacy of intrathecal delivery of GC101 gene therapy drug as a treatment of spinal muscular atrophy Type 1 (SMA 1) patients.
Diagnosis of SMA based on gene mutation analysis with bi-allelic SMN1 mutations (deletion or point mutations) and 2 copies of SMN2;
Onset of disease before 6 months of age
The patient's legal guardian(s) must be able to understand the purpose and risks of the study and voluntarily provide signed and dated informed consent prior to any study-related procedures being performed.
Patient who has participated in a previous gene therapy research trials;
Patient who has received Nusinersen and Risdiplam treatment;
Patient who has AAV9 neutralizing antibody titer ≥1:200;
Patient who requires non-invasive ventilatory support averaging≥16 hours/day;
GeneCradle Inc
Lead sponsor