DRUGapprovedgene therapy
intrathecal onasemnogene abeparvovec
Brand names: Itvisma, OAV101 IT
Mechanism
AAV9-delivered SMN1 gene replacement, single intrathecal injection. Non-IV route decouples dose from body weight (vs Zolgensma IV which is BW-limited).
Approved indications
SMA biallelic SMN1 (age ≥2)children ≥2teensadults
Related claims (3)
| Type | Predicate | Conf | Source |
|---|---|---|---|
| drug efficacy | Intrathecal onasemnogene abeparvovec administration, at fixed, low doses, may enable treatment of heavier patients ineligible for weight-based intravenous dosing. | 59% | 36911944 |
| drug efficacy | Intrathecal onasemnogene abeparvovec (OAV101 IT) may enable a one-time gene transfer therapy, addressing an unmet need across the broader spinal muscular atrophy (SMA) population. | 51% | 41360995 |
| safety | Intrathecal onasemnogene abeparvovec was safe and well-tolerated (clinical trial result). | 45% | 36911944 |
Off-Target Findings (0)
No Boltz-2 / Chai-1 off-target panel claims recorded for this drug.