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

Prior treatment

• 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.

Other illness

• 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.

Lab values

• Clinically significant abnormal laboratory values prior to administration.

Age

• Weight-for-age below the 3rd percentile for the same sex and age based on WHO Child Growth Standards.

Other

• Pulse oximetry \< 96% saturation at screening while the patient is awake or asleep without any supplemental oxygen or respiratory support.

• 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.