4 assets in Spinal Muscular Atrophy, Type 1

Therapeutic assets addressing Spinal Muscular Atrophy, Type 1, rated under Floatz methodology v0.2 and sorted by current rating.

Asset	Sponsor	Phase	Rating
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 1	Novartis Gene Therapies	P3	BB (69)
exg001-307 Spinal Muscular Atrophy, Type 1	Guangzhou Jiayin Biotech Ltd	P1/2	CCC (54)
gb221 Spinal Muscular Atrophy, Type 1	Gemma Biotherapeutics	P1/2	CCC (48)
skg0201 Spinal Muscular Atrophy, Type 1	Lanyue Biotech (Hangzhou) Co., Ltd.	P1/2	CCC (48)