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Gcb-001

Spinal Muscular Atrophy, Type II

Genecombio Ltd.UNKNOWNN/AActive
D
30/ 100
Provisional rating · Methodology v0.2

Rating breakdown

Clinical Evidence
15/ 100
Competitive Position
89/ 100

Competitive context

6 Active assets in the Spinal Muscular Atrophy, Type II cohort.

Asset / SponsorPhaseRating
Apitegromab
Scholar Rock, Inc.
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 3B
Olesoxime, E-
Hoffmann-La Roche
Phase 2B
Risdiplam
Scholar Rock, Inc.
Phase 2CCC
Nusinersen
Scholar Rock, Inc.
Phase 2CCC
Sodium Phenylbutyrate
Westat
Phase 1/2CCC
gc101
GeneCradle Inc
Phase 1/2CCC
oav101
Novartis Pharmaceuticals
Phase 3NR

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