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vesemnogene lantuparvovec
- Development stage
- Phase 3
- Lead developer
- Lantus Biopharma
- Modality
- Gene Therapies
- Administration
- Intravenous, Intrathecal
01
Overview
Vesemnogene lantuparvovec is a gene therapy, specifically an adeno-associated virus (AAV) vector carrying the human survival motor neuron 1 (SMN1) gene. It is being developed by Lantu Biopharma for the treatment of Spinal Muscular Atrophy (SMA). The therapy aims to address the genetic defect in SMA patients by delivering a functional SMN1 gene.
02
Targets
SMN (Survival motor neuron protein)
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