SMA – THERAPY: P.255 One-time administration of AVXS-101 intrathecal (IT) for spinal muscular atrophy in the phase 1 study (STRONG): safety report

Abstract

Spinal muscular atrophy (SMA) is caused by deletion/mutation of the survival motor neuron 1 gene (SMN1). AVXS-101 IT is a one-time gene therapy designed to deliver fully functional copy of human SMN gene to address the genetic root cause of SMA. STRONG (CL-102, NCT03381729), a phase 1, open-label study, assesses the safety, optimal dose, and efficacy of AVXS-101 IT. Patients (pts; biallelic SMN1 loss, 3xSMN2) aged ≥6<60 months who could sit, but not stand/walk received a one-time AVXS-101 IT dose (dose A: 6.0 × 10e13 vg; B: 1.2 × 10e14 vg; C: 2.4 × 10e14 vg).