Gene Therapy Services for Spinal Muscular Atrophy (Zolgensma® and OAV101)

This opportunity involves providing two gene therapy services for patients with spinal muscular atrophy (SMA). The first service covers Onasemnogene abeparvovec intravenous (Zolgensma®), a one-time gene replacement therapy for infants aged 12 months and under. Zolgensma® uses an adeno-associated virus vector to deliver a functional copy of the SMN1 gene directly into motor neurons and peripheral tissues, enabling production of survival motor neuron (SMN) protein that is deficient in SMA patients. The therapy is administered intravenously and provides sustained therapeutic benefit. The second service covers Onasemnogene abeparvovec intrathecal (OAV101 IT), a one-time gene therapy for SMA patients aged 2 years and over, delivered via intrathecal injection to treat a separate patient population. OAV101 IT is undergoing NICE technology appraisal review with expected guidance publication in late 2026. Suppliers should be prepared to deliver both intravenous and intrathecal administration services, patient management protocols, and regulatory compliance aligned with NICE recommendations.