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Adipose Derived Mesenchymal Stem Cell

Infantile Spinal Muscular Atrophy, Type I [Werdnig- Hoffman]

Tehran University of Medical SciencesCell therapyPhase 1/2Dormant
CCC
48/ 100
Provisional rating · Methodology v0.2

Rating breakdown

Clinical Evidence
33/ 100
Competitive Position
95/ 100

Competitive context

5 Active assets in the Infantile Spinal Muscular Atrophy, Type I [Werdnig- Hoffman] cohort.

Asset / SponsorPhaseRating
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)
Novartis Gene Therapies
Phase 3BB
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)
Novartis Gene Therapies
Phase 3BB
exg001-307
Guangzhou Jiayin Biotech Ltd
Phase 1/2CCC
Valproic Acid
University of Utah
Phase 1/2CCC
Levocarnitine
University of Utah
Phase 1/2CCC

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