Floatz Rating
C36/100
Confidence
Indicative
Sponsor
EdiGene (GuangZhou) Inc.
Modality
UNKNOWN
Development Phase
Phase 1
Status
Active
Evidence ledger · v0.2
Clinical Evidence
Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
21Moderate confidence
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT05752123CT.gov A Study to Evaluate the Safety and Efficacy of ET-01 Transplantation in Subjects with Transfusion Dependent Β-Thalassaemia. | — | Terminated | — | — | — |
NCT04390971CT.gov Safety and Efficacy Evaluation of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassaemia | — | Completed | — | — | — |
NCT04925206CT.gov A Safety and Efficacy Study Evaluating ET-01 in Subjects with Transfusion Dependent Β-Thalassaemia | — | Terminated | — | — | — |
Competitive Position
Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
95Indicative
Detailed axis rationale is planned and will be published soon.
Same indication · Beta-Thalassemia Major
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| et-01 (this asset) | EdiGene (GuangZhou) Inc. | P1 | C · 36 |
| Deferasirox | Pakistan Blood and Marrow Transplant (PBMT) Group | P4 | BBB |
| Hydroxyurea | Bahria University | P4 | BBB |
| Deferiprone | Pakistan Blood and Marrow Transplant (PBMT) Group | P4 | BBB |
| Desferrioxamine B | Pakistan Blood and Marrow Transplant (PBMT) Group | P4 | BBB |
| Tacrolimus | M.D. Anderson Cancer Center | P4 | BBB |
| Fludarabine | M.D. Anderson Cancer Center | P4 | BBB |
| Busulfanum | M.D. Anderson Cancer Center | P4 | BBB |
| Cyclophosphamide | M.D. Anderson Cancer Center | P4 | BB |
+42 more in the Beta-Thalassemia Major 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. et-01 in Beta-Thalassemia Major. 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/et-01-beta-thalassemia-major
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