Autologous Cd34+ Cells Encoding .Beta.A-T87Q-Globin Gene
Beta Thalassemia
Explore 35 assets in Beta Thalassemia →Floatz Rating
B60/100
Confidence
Indicative
Sponsor
CorrectSequence Therapeutics Co., Ltd
Modality
Cell 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.
49High confidence
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT03207009CT.gov A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia | — | Completed | — | — | — |
NCT02906202CT.gov A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia, Who do Not Have a β0/β0 Genotype | — | 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 · Beta Thalassemia
+42 more in the Beta Thalassemia cohort
Other indications for Autologous Cd34+ Cells Encoding .Beta.A-T87Q-Globin Gene
| Indication | Sponsor | Phase | Rating |
|---|---|---|---|
| Beta-Thalassemia Major | — | P1/2 | CCC · 55 |
| Sickle Cell Disease | — | P1/2 | CC · 44 |
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.
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Citation
Floatz Terminal. Autologous Cd34+ Cells Encoding .Beta.A-T87Q-Globin Gene in Beta Thalassemia. 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/autologous-cd34-cells-encoding-betaa-t87q-globin-gene-beta-thalassemia
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