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 III
Novartis Gene Therapiesgene_therapyPhase 3Active
B
60/ 100
Provisional rating · Methodology v0.2
Rating breakdown
- Clinical Evidence
- 45/ 100
- Competitive Position
- 95/ 100
Competitive context
4 Active assets in the Spinal Muscular Atrophy, Type III cohort.
| Asset / Sponsor | Phase | Rating |
|---|---|---|
Apitegromab Scholar Rock, Inc. | Phase 3 | BB |
Pyridostigmine UMC Utrecht | Phase 2 | B |
Olesoxime, E- Hoffmann-La Roche | Phase 2 | B |
AMIFAMPRIDINE Catalyst Pharmaceuticals, Inc. | Phase 2 | CCC |
Risdiplam Scholar Rock, Inc. | Phase 2 | CCC |
Nusinersen Scholar Rock, Inc. | Phase 2 | CCC |
Sodium Phenylbutyrate Westat | Phase 1/2 | CCC |
gc101 GeneCradle Inc | Phase 1/2 | CC |
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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