Floatz Rating
BB67/100
Confidence
Indicative
Sponsor
CSL Behring
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.
56High confidence
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT07080905CT.gov Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (≥ 12 to < 18 Years of Age) With Severe or Moderately Severe Hemophilia B | — | Recruiting | — | — | — |
NCT06003387CT.gov Efficacy and Safety of CSL222 (Etranacogene Dezaparvovec) Gene Therapy in Adults With Hemophilia B With Pretreatment Adeno-associated Virus Serotype 5 (AAV5) Neutralizing Antibodies (Nabs) | — | Recruiting | — | — | — |
NCT05962398CT.gov Long-term Follow-up Study of Male Adults With Hemophilia B Previously Treated With Etranacogene Dezaparvovec (CSL222) | — | Enrolling By Invitation | — | — | — |
NCT06008938CT.gov An Observational Cohort Study to Characterize the Effectiveness and Safety of HEMGENIX® in Patients With Hemophilia B | — | Recruiting | — | — | — |
NCT03489291CT.gov Dose Confirmation Trial of AAV5-hFIXco-Padua | — | Completed | — | — | — |
NCT03569891CT.gov HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients | — | Completed | — | — | — |
Competitive Position
Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
89Indicative
Detailed axis rationale is planned and will be published soon.
Same indication · Hemophilia B
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Etranacogene Dezaparvovec (this asset) | CSL Behring | P3 | BB · 67 |
| 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. Etranacogene Dezaparvovec 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/etranacogene-dezaparvovec-hemophilia-b
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