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Adipose Derived Mesenchymal Stem Cell

Spinal Muscular Atrophy, Type 1

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

4 Active assets in the Spinal Muscular Atrophy, Type 1 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
exg001-307
Guangzhou Jiayin Biotech Ltd
Phase 1/2CCC
Valproic Acid
University of Utah
Phase 1/2CCC
Levocarnitine
University of Utah
Phase 1/2CCC
gb221
Gemma Biotherapeutics
Phase 1/2CCC
skg0201
Lanyue Biotech (Hangzhou) Co., Ltd.
Phase 1/2CCC
Sodium Phenylbutyrate
Westat
Phase 1/2CCC

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