Floatz Rating
CC47/100
Confidence
Indicative
Sponsor
Biocad
Modality
UNKNOWN
Development Phase
Phase 1/2
Status
Active
Evidence ledger · v0.2
Clinical Evidence
Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
33Indicative
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT05747261CT.gov Study of the Safety and Efficacy of an Adeno-Associated Viral Vector Carrying the SMN Gene After a Single Intravenous Administration of Escalating Doses in Children With Spinal Muscular Atrophy (BLUEBELL) | — | Recruiting | — | — | — |
Competitive Position
Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
89Moderate confidence
Detailed axis rationale is planned and will be published soon.
Same indication · Spinal Muscular Atrophy
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Anb-004 (this asset) | Biocad | P1/2 | CC · 47 |
| Nusinersen | Scholar Rock, Inc. | P4 | A |
| Risdiplam | Scholar Rock, Inc. | P4 | A |
| oav101 | Novartis Pharmaceuticals | P4 | BBB |
| 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 Pharmaceuticals | P4 | BBB |
| Apitegromab | Scholar Rock, Inc. | P3 | BB |
| Hydroxyurea | Kaohsiung Medical University Chung-Ho Memorial Hospital | P2/3 | BB |
| Vesemnogene Lantuparvovec | Lantu Biopharma | P3 | BB |
| Levocarnitine | All India Institute of Medical Sciences | P3 | BB |
+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
- NusinersenScholar Rock, Inc.A
- RisdiplamScholar Rock, Inc.A
- oav101Novartis PharmaceuticalsBBB
- 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 PharmaceuticalsBBB
- ApitegromabScholar Rock, Inc.BB
Citation
Floatz Terminal. Anb-004 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/anb-004-spinal-muscular-atrophy
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