OAV-101

OAV-101 is an investigational intrathecal formulation of onasemnogene abeparvovec being developed by Novartis for spinal muscular atrophy, particularly older pediatric patients with SMA who are not well served by systemic intravenous gene transfer. It is an AAV9-mediated gene therapy and biologic designed to deliver a functional copy of the **survival motor neuron 1** gene to motor neurons after direct administration into the cerebrospinal fluid, with the goal of restoring SMN protein expression and modifying the underlying genetic cause of disease. OAV-101 has been studied as a one-time treatment in nonambulatory patients with SMA Type 2, including in the Phase 3 STEER program, and represents the intrathecal development program related to the same core active construct used in Zolgensma.

Brand names

Itvisma

Other names

onasemnogene abeparvovec