Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
TSHA-101 · 1 trial · 1 indication
Incidence, severity, and relatedness of TEAEs
Number of participants with Changes from Baseline in laboratory assessments
Changes from Baseline in 12-lead ECG findings in QT interval
| Arm | Type | Description |
|---|---|---|
| TSHA-101 | EXPERIMENTAL | Subjects who will receive one-time intrathecal TSHA-101, brain volume based sliding scale for dosage |
| Name | Type | Description |
|---|---|---|
| TSHA-101 | BIOLOGICAL | AAV9 viral vector containing HEXA and HEXB genes to be administered via Intrathecal injection |
Key Inclusion Criteria: * male or female with age less than or equal to 15 months * diagnosis of GM2 gangliosidosis with genetic and enzymatic documentation of infantile disease Key Exclusion Criteria: * a second neurodevelopmental disorder independent of the HEXA or HEXB * inability to tolerate ...