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AVXS-101 IT (onasemnogene abeparvovec)
- Development stage
- Phase 3
- Lead developer
- Novartis
- Modality
- Gene Therapies
- Administration
- Intrathecal
01
Overview
AVXS-101 IT (also known as OAV101) is an intrathecal formulation of the gene therapy onasemnogene abeparvovec, developed for the treatment of spinal muscular atrophy (SMA). It utilizes a non-replicating adeno-associated virus serotype 9 (AAV9) vector to deliver a functional copy of the human survival motor neuron (SMN) gene directly into the cerebrospinal fluid. This intrathecal delivery is designed to target motor neurons in the central nervous system more efficiently in older, non-ambulatory patients (typically Type 2 SMA) compared to intravenous administration. By restoring SMN protein levels, the therapy aims to halt disease progression and improve motor milestones. The program was initiated by AveXis and is currently being advanced by Novartis.
- Brand names
- Zolgensma
- Other names
- OAV101 ITOAV-101 ITOAV 101 ITintrathecal onasemnogene abeparvovec
02
Targets
SMN (Survival motor neuron protein)
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