Autologous Cd34+ Cells Encoding .Beta.A-T87Q-Globin Gene
Beta-Thalassemia Major
Explore 31 assets in Beta-Thalassemia Major →Floatz Rating
CCC55/100
Confidence
Indicative
Sponsor
Genetix Biotherapeutics Inc.
Modality
Cell therapy
Development Phase
Phase 1/2
Status
Dormant
Evidence ledger · v0.2
Clinical Evidence
Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
42High confidence
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT01745120CT.gov A Study Evaluating the Safety and Efficacy of the LentiGlobin BB305 Drug Product in β-Thalassemia Major Participants | — | Completed | — | — | — |
NCT02151526CT.gov A Study Evaluating the Safety and Efficacy of LentiGlobin BB305 Drug Product in β-Thalassemia Major (Also Referred to as Transfusion-dependent β-Thalassemia [TDT]) and Sickle Cell Disease | — | Completed | — | — | — |
Competitive Position
Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
95High confidence
Detailed axis rationale is planned and will be published soon.
Same indication · Beta-Thalassemia Major
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Autologous Cd34+ Cells Encoding .Beta.A-T87Q-Globin Gene (this asset) | Genetix Biotherapeutics Inc. | P1/2 | CCC · 55 |
| 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 |
| Tacrolimus | M.D. Anderson Cancer Center | P4 | BBB |
| Desferrioxamine B | Pakistan Blood and Marrow Transplant (PBMT) Group | 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
Other indications for Autologous Cd34+ Cells Encoding .Beta.A-T87Q-Globin Gene
| Indication | Sponsor | Phase | Rating |
|---|---|---|---|
| Beta Thalassemia | — | P3 | B · 60 |
| 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.
Related assets
Citation
Floatz Terminal. Autologous Cd34+ Cells Encoding .Beta.A-T87Q-Globin Gene 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/autologous-cd34-cells-encoding-betaa-t87q-globin-gene-beta-thalassemia-major
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