A Phase I/II Clinical Study to Evaluate SKG0201 Injection in Subjects With Spinal Muscular Atrophy Type I

This is a multicenter, open-label, dose-escalation phase I/II clinical study to evaluate the safety, preliminary efficacy and immunogenicity of SKG0201 injection in subjects with Spinal Muscular Atrophy Type I (SMA I).

Inclusion criteria

• Type I SMA, defined by bi-allelic mutations in the SMN1 gene.
• Clinical history and signs are consistent with type I SMA, such as hypotonia, delayed motor function development, and poor head control.
• On the day of administration, the age of the subjects do not exceed 180 days after birth.

Exclusion criteria

• Pulse oximetry < 96% saturation at screening while the patient is awake or asleep without any supplemental oxygen or respiratory support.
• Weight-for-age below the 3rd percentile for the same sex and age based on WHO Child Growth Standards.
• Active viral infection.
• In the presence of other severe infections or diseases that require systemic anti-infection treatment.
• Known allergy to prednisolone, other glucocorticoids, or SKG0201's excipients.
• Clinically significant abnormal laboratory values prior to administration.
• Previously used Zolgensma or other SMA gene therapy drugs, or currently participating in other SMA clinical studies on therapeutic drugs.
• Having previously undergone major surgery or expected to undergo major surgery during the study assessment period.