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All-P-Ambo-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thiocytidylyl-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]Adenylyl-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thio[(5-Methyl)Cytidylyl)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-[(5-Methyl)Uridylyl-)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thio[(5-Methyl)Uridylyl)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thio[(5-Methyl)Uridylyl)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thio[(5-Methyl)Cytidylyl)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thioadenylyl)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thio[(5-Methyl)Uridylyl)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thioadenylyl)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thioadenylyl)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thio[(5-Methyl)Uridylyl)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thioguanylyl)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thio[(5-Methyl)Cytidylyl)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thio[(5-Methyl)Uridylyl)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thioguanylyl)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thioguanylyl)]-(5-Methyl)Cytidine

Spinal Muscular Atrophy

Explore 20 assets in Spinal Muscular Atrophy →
Floatz Rating
B60/100
Confidence
Indicative
v0.2
Sponsor
Biogen
Modality
nucleic_acid
Development Phase
Phase 3
Status
Active
Evidence ledger · v0.2

Clinical Evidence

Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
45Moderate confidence
Detailed axis rationale is planned and will be published soon.
TrialPhaseStatusNPrimary endpointReadout
NCT07444450CT.gov
A Study to Learn About the Safety and Effects of Salanersen (BIIB115) When Given to Babies With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec
—Not Yet Recruiting———
NCT07221669CT.gov
A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety When Given Before Symptoms Appear in Babies With Genetically Diagnosed Spinal Muscular Atrophy (SMA)
—Recruiting———
NCT07444476CT.gov
A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety in Participants Aged 15 to 60 Years With Spinal Muscular Atrophy (SMA) Who Are Either New to SMA Treatment or Were Previously Treated With Risdiplam
—Recruiting———

Competitive Position

Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
95High confidence
Detailed axis rationale is planned and will be published soon.

Same indication · Spinal Muscular Atrophy

AssetSponsorPhaseRating
All-P-Ambo-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thiocytidylyl-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]Adenylyl-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thio[(5-Methyl)Cytidylyl)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-[(5-Methyl)Uridylyl-)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thio[(5-Methyl)Uridylyl)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thio[(5-Methyl)Uridylyl)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thio[(5-Methyl)Cytidylyl)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thioadenylyl)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thio[(5-Methyl)Uridylyl)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thioadenylyl)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thioadenylyl)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thio[(5-Methyl)Uridylyl)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thioguanylyl)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thio[(5-Methyl)Cytidylyl)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thio[(5-Methyl)Uridylyl)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thioguanylyl)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thioguanylyl)]-(5-Methyl)Cytidine (this asset)BiogenP3B · 60
NusinersenScholar Rock, Inc.P4A
RisdiplamScholar Rock, Inc.P4A
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 PharmaceuticalsP4BBB
oav101Novartis PharmaceuticalsP4BBB
ApitegromabScholar Rock, Inc.P3BB
HydroxyureaKaohsiung Medical University Chung-Ho Memorial HospitalP2/3BB
Vesemnogene LantuparvovecLantu BiopharmaP3BB
LevocarnitineAll India Institute of Medical SciencesP3BB

+30 more in the Spinal Muscular Atrophy cohort

Scientific Foundation

Strength of the underlying biology: target validation, tractability, modality fit, and how related mechanisms have fared.
NR

Planned for methodology v0.2.

Development Feasibility

How realistically the program can be executed, drawing on modality precedent, enrollment dynamics, and sponsor delivery.
NR

Planned for methodology v0.2.

Commercial Opportunity

Commercial prize: addressable population, unmet need, and the value case for the indication.
NR

Planned for methodology v0.2.

IP & Exclusivity

Exclusivity position, covering patent protection and freedom-to-operate runway.
NR

Planned for methodology v0.2.

Manufacturing & Supply

Manufacturing and supply readiness, driven by modality process and scale-up risk.
NR

Planned for methodology v0.2.

Related assets

Citation

Floatz Terminal. All-P-Ambo-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thiocytidylyl-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]Adenylyl-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thio[(5-Methyl)Cytidylyl)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-[(5-Methyl)Uridylyl-)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thio[(5-Methyl)Uridylyl)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thio[(5-Methyl)Uridylyl)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thio[(5-Methyl)Cytidylyl)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thioadenylyl)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thio[(5-Methyl)Uridylyl)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thioadenylyl)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thioadenylyl)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thio[(5-Methyl)Uridylyl)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thioguanylyl)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thio[(5-Methyl)Cytidylyl)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thio[(5-Methyl)Uridylyl)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thioguanylyl)]-(5'?3')-[2?-O-(2-Methylamino)-2-Oxoethyl]-P-Thioguanylyl)]-(5-Methyl)Cytidine in Spinal Muscular Atrophy. Methodology v0.2.
Rated under v0.2 effective July 8, 2026. Last refreshed July 8, 2026.
Accessed July 14, 2026.
https://terminal.floatz.ai/assets/all-p-ambo-53-2-o-2-methylamino-2-oxoethyl-p-thiocytidylyl-53-2-o-2-methylamino-2-oxoethyladenylyl-53-2-o-2-methylamino-2-oxoethyl-p-thio5-methylcytidylyl-53-2-o-2-methylamino-2-oxoethyl-5-methyluridylyl-53-2-o-2-methylamino-2-oxoethyl-p-thio5-methyluridylyl-53-2-o-2-methylamino-2-oxoethyl-p-thio5-methyluridylyl-53-2-o-2-methylamino-2-oxoethyl-p-thio5-methylcytidylyl-53-2-o-2-methylamino-2-oxoethyl-p-thioadenylyl-53-2-o-2-methylamino-2-oxoethyl-p-thio5-methyluridylyl-53-2-o-2-methylamino-2-oxoethyl-p-thioadenylyl-53-2-o-2-methylamino-2-oxoethyl-p-thioadenylyl-53-2-o-2-methylamino-2-oxoethyl-p-thio5-methyluridylyl-53-2-o-2-methylamino-2-oxoethyl-p-thioguanylyl-53-2-o-2-methylamino-2-oxoethyl-p-thio5-methylcytidylyl-53-2-o-2-methylamino-2-oxoethyl-p-thio5-methyluridylyl-53-2-o-2-methylamino-2-oxoethyl-p-thioguanylyl-53-2-o-2-methylamino-2-oxoethyl-p-thioguanylyl-5-methylcytidine-spinal-muscular-atrophy

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