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exg001-307

Spinal Muscular Atrophy, Type 1

Guangzhou Jiayin Biotech LtdUNKNOWNPhase 1/2Active
CCC
54/ 100
Provisional rating · Methodology v0.2

Rating breakdown

Clinical Evidence
40/ 100
Competitive Position
95/ 100

Competitive context

3 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
Levocarnitine
University of Utah
Phase 1/2CCC
Valproic Acid
University of Utah
Phase 1/2CCC
gb221
Gemma Biotherapeutics
Phase 1/2CCC
Sodium Phenylbutyrate
Westat
Phase 1/2CCC
skg0201
Lanyue Biotech (Hangzhou) Co., Ltd.
Phase 1/2CCC
Adipose Derived Mesenchymal Stem Cell
Tehran University of Medical Sciences
Phase 1/2CCC

Same molecule, other indications

exg001-307 is also rated in:

spinal muscular atrophyActive
C
Indicative · Early Phase 1
39 / 100
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