Floatz Rating
BB64/100
Confidence
Indicative
Sponsor
Lantu Biopharma
Modality
gene_therapy
Development Phase
Phase 3
Status
Active
Evidence ledger · v0.2
Clinical Evidence
Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
52Moderate confidence
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT07265232CT.gov Real World Clinical Effectiveness & Safety of Vesemnogene Lantuparvovec for Spinal Muscular Atrophy (SMA) in Low-middle Income Countries (LMIC). | — | Recruiting | — | — | — |
NCT06288230CT.gov An Open Label Study of Gene Therapy Product (Vesemnogene Lantuparvovec) in Spinal Muscular Atrophy | — | Active Not Recruiting | — | — | — |
Competitive Position
Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
89High confidence
Detailed axis rationale is planned and will be published soon.
Same indication · Spinal Muscular Atrophy
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Vesemnogene Lantuparvovec (this asset) | Lantu Biopharma | P3 | BB · 64 |
| Nusinersen | Scholar Rock, Inc. | P4 | A |
| Risdiplam | Scholar Rock, Inc. | P4 | A |
| 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 |
| oav101 | Novartis Pharmaceuticals | P4 | BBB |
| Apitegromab | Scholar Rock, Inc. | P3 | BB |
| Hydroxyurea | Kaohsiung Medical University Chung-Ho Memorial Hospital | P2/3 | BB |
| Levocarnitine | All India Institute of Medical Sciences | P3 | BB |
| Valproic Acid | All India Institute of Medical Sciences | P3 | B |
+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
- 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
- oav101Novartis PharmaceuticalsBBB
- ApitegromabScholar Rock, Inc.BB
Citation
Floatz Terminal. Vesemnogene Lantuparvovec 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/vesemnogene-lantuparvovec-spinal-muscular-atrophy
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