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
A
80/ 100
Provisional rating · Methodology v0.2
Rating breakdown
- Clinical Evidence
- 74/ 100
- Competitive Position
- 89/ 100
Competitive context
21 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 |
Srk-015 Scholar Rock, Inc. | Phase 3 | BB |
Hydroxyurea Kaohsiung Medical University Chung-Ho Memorial Hospital | Phase 2/3 | BB |
View all 40 →
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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