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)
Gene Therapy
Novartis Gene Therapiesgene_therapyPhase 3Dormant
B
62/ 100
Provisional rating · Methodology v0.2
Rating breakdown
- Clinical Evidence
- 49/ 100
- Competitive Position
- 89/ 100
Competitive context
4 Active assets in the Gene Therapy cohort.
| Asset / Sponsor | Phase | Rating |
|---|---|---|
Spk-8016 Spark Therapeutics, Inc. | Phase 1/2 | CCC |
Valoctocogene Roxaparvovec BioMarin Pharmaceutical | Phase 1/2 | CC |
Kln-1010 Kelonia Therapeutics, Inc. | Phase 1 | CC |
bmd003 Peking Union Medical College Hospital | Early Phase 1 | C |
Clozapine Ruijin Hospital | Early Phase 1 | C |
Of Gs001 Institute of Hematology & Blood Diseases Hospital, China | N/A | D |
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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