Non-Replicating Recombinant Self-Complementary (Sc) Adeno-Associated Virus Serotype 9 (Aav9) Vector Containing The Cdna Of The Survival Of Motor Neuron 2 (Smn2) Gene Under The Control Of The Hybrid Cytomegalovirus (Cmv) Enhancer/Chicken Beta-Actin Promoter (Cba)
Spinal Muscular Atrophy, Type 1
Novartis Gene Therapiesgene_therapyPhase 3Active
BB
69/ 100
Provisional rating · Methodology v0.2
Rating breakdown
- Clinical Evidence
- 56/ 100
- Competitive Position
- 95/ 100
Competitive context
3 Active assets in the Spinal Muscular Atrophy, Type 1 cohort.
| Asset / Sponsor | Phase | Rating |
|---|---|---|
exg001-307 Guangzhou Jiayin Biotech Ltd | Phase 1/2 | CCC |
Levocarnitine University of Utah | Phase 1/2 | CCC |
Valproic Acid University of Utah | Phase 1/2 | CCC |
gb221 Gemma Biotherapeutics | Phase 1/2 | CCC |
Sodium Phenylbutyrate Westat | Phase 1/2 | CCC |
skg0201 Lanyue Biotech (Hangzhou) Co., Ltd. | Phase 1/2 | CCC |
Adipose Derived Mesenchymal Stem Cell Tehran University of Medical Sciences | Phase 1/2 | CCC |
Same molecule, other indications
Non-Replicating Recombinant Self-Complementary (Sc) Adeno-Associated Virus Serotype 9 (Aav9) Vector Containing The Cdna Of The Survival Of Motor Neuron 2 (Smn2) Gene Under The Control Of The Hybrid Cytomegalovirus (Cmv) Enhancer/Chicken Beta-Actin Promoter (Cba) is also rated in:
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