Novartis Gene Therapies
Quick facts
Marketed products
- Onasemnogene Abeparvovec-xioi · Neurology
Onasemnogene abeparvovec-xioi is a gene therapy that delivers a functional copy of the SMN1 gene via an adeno-associated virus vector to restore survival motor neuron protein production in patients with spinal muscular atrophy.
Phase 1 pipeline
- AVXS-101 · Rare Disease
Gene therapy for spinal muscular atrophy
Competitive intelligence
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