Floatz Rating
B60/100
Confidence
Indicative
Sponsor
Institute of Hematology & Blood Diseases Hospital, China
Modality
gene_therapy
Development Phase
Phase 2/3
Status
Active
Evidence ledger · v0.2
Clinical Evidence
Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
49Moderate confidence
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT05709288CT.gov Gene Therapy for Hemophilia B Patients Aged 12-18 Years Old | — | Recruiting | — | — | — |
NCT05203679CT.gov Evaluation of the Safety and Efficacy of Hemophilia B Gene Therapy Drug | — | Active Not Recruiting | — | — | — |
NCT04135300CT.gov Gene Therapy for Chinese Hemophilia B | — | Completed | — | — | — |
Competitive Position
Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
81High confidence
Detailed axis rationale is planned and will be published soon.
Same indication · Hemophilia B
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Bbm-H901 (this asset) | Institute of Hematology & Blood Diseases Hospital, China | P2/3 | B · 60 |
| Factor Ix | CSL Behring | P4 | A |
| Eptacog Alfa (Activated) | AryoGen Pharmed Co. | P4 | A |
| Nonacog Alfa | Pfizer | P4 | A |
| Nonacog Beta Pegol | Novo Nordisk A/S | P3 | A |
| Coagulation Factor Viia | American Thrombosis and Hemostasis Network | P4 | BBB |
| Albutrepenonacog Alfa | CSL Behring | P3 | BBB |
| Fitusiran | Genzyme, a Sanofi Company | P3 | BBB |
| Bax 326 | Baxalta now part of Shire | P3 | BB |
+40 more in the Hemophilia B 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. Bbm-H901 in Hemophilia B. 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/bbm-h901-hemophilia-b
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