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7 assets in Spinal Muscular Atrophy, Type II

Therapeutic assets addressing Spinal Muscular Atrophy, Type II, rated under Floatz methodology v0.2 and sorted by current rating.

AssetSponsorPhaseRating
Apitegromab
Spinal Muscular Atrophy, Type II
Scholar Rock, Inc.P3BB (67)
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)
Spinal Muscular Atrophy, Type II
Novartis Gene TherapiesP3B (60)
Nusinersen
Spinal Muscular Atrophy, Type II
Scholar Rock, Inc.P2CCC (53)
Risdiplam
Spinal Muscular Atrophy, Type II
Scholar Rock, Inc.P2CCC (53)
gc101
Spinal Muscular Atrophy, Type II
GeneCradle IncP1/2CCC (48)
Gcb-001
Spinal Muscular Atrophy, Type II
Genecombio Ltd.N/AD (30)
oav101
Spinal Muscular Atrophy, Type II
Novartis PharmaceuticalsP3NR ()