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onasemnogene abeparvovec
- Development stage
- Approved
- Lead developer
- Novartis
- Modality
- Gene Therapies, Recombinant Proteins and Enzymes
- Administration
- Intravenous
01
Overview
Onasemnogene abeparvovec is an adeno-associated virus serotype 9 (AAV9) vector-based gene therapy designed to treat spinal muscular atrophy (SMA), a rare neuromuscular disorder caused by bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. The therapy delivers a functional copy of the *SMN* gene to motor neurons, enabling production of the SMN protein that is deficient in SMA patients. This one-time intravenous infusion aims to halt disease progression and improve or preserve motor function in affected children, particularly those under two years old. Onasemnogene abeparvovec was developed by AveXis and is marketed as Zolgensma by Novartis[1][3][4][5][6][8].
- Brand names
- Zolgensma
- Other names
- onasemnogene abeparvovec-xioi
02
Targets
SMN (Survival motor neuron protein)
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