Tender: Onasemnogene Abeparvovec (Zolgensma®) IV for Infants and OAV101 Intrathecal for Older SMA Patients – Gene Therapy Services
Released 5 months ago
About
Supplier needed for onasemnogene abeparvovec intravenous (Zolgensma®) and onasemnogene abeparvovec intrathecal (OAV101 IT) services. Onasemnogene abeparvovec intravenous (Zolgensma®) is a one-time, in vivo gene replacement therapy for babies aged 12 months and under that uses an AAV9 vector modified to carry a functional copy of the human SMN1 gene; it is administered intravenously to deliver the gene into motor neurones and peripheral tissues and enable sustained expression of SMN protein. Onasemnogene abeparvovec intrathecal (OAV101 IT) is a one-time in vivo gene replacement therapy proposed to treat individuals with spinal muscular atrophy (SMA) aged 2 years and over by intrathecal injection; it targets a distinct cohort to Zolgensma and will be assessed via NICE’s technology appraisal programme with expected final guidance publication in late 2026.
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