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
Novartis Pharmaceuticalsgene_therapyPhase 4 / ApprovedActive
BBB
78/ 100
Provisional rating · Methodology v0.2
Rating breakdown
- Clinical Evidence
- 74/ 100
- Competitive Position
- 89/ 100
Competitive context
19 Active assets in the Spinal Muscular Atrophy cohort.
| Asset / Sponsor | Phase | Rating |
|---|---|---|
Nusinersen Scholar Rock, Inc. | Phase 4 | A |
Risdiplam Scholar Rock, Inc. | Phase 4 | A |
oav101 Novartis Pharmaceuticals | Phase 4 | BBB |
Apitegromab Scholar Rock, Inc. | Phase 3 | BB |
Hydroxyurea Kaohsiung Medical University Chung-Ho Memorial Hospital | Phase 2/3 | BB |
View all 38 →
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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