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 |
|---|---|---|
Srk-015 Scholar Rock, Inc. | Phase 3 | BB |
Pyridostigmine UMC Utrecht | Phase 2 | B |
OLESOXIME, E- Hoffmann-La Roche | Phase 2 | B |
Pyridostigmine Centre Hospitalier Régional de la Citadelle | Phase 2 | B |
Nusinersen Scholar Rock, Inc. | Phase 2 | CCC |
View all 9 →
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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