Autologous Cd34+ Hsc Cells Transduced With The Lentiviral Vector Containing A Shrna Targeting Bcl11A
Sickle Cell Disease
Explore 133 assets in Sickle Cell Disease →Floatz Rating
CCC52/100
Confidence
Indicative
Sponsor
David Williams
Modality
gene_therapy
Development Phase
Phase 2
Status
Active
Evidence ledger · v0.2
Clinical Evidence
Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
45Moderate confidence
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT05353647CT.gov A Gene Transfer Study Inducing Fetal Hemoglobin in Sickle Cell Disease (GRASP, BMT CTN 2001) | — | Active Not Recruiting | — | — | — |
NCT03282656CT.gov Gene Transfer for Sickle Cell Disease | — | Active Not Recruiting | — | — | — |
NCT07640815CT.gov Expanded Access Protocol for Gene Therapy Utilizing shmiR Lentivirus Vector to Induce Fetal Hemoglobin in Sickle Cell Disease | — | Temporarily Not Available | — | — | — |
Competitive Position
Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
65Indicative
Detailed axis rationale is planned and will be published soon.
Same indication · Sickle Cell Disease
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Autologous Cd34+ Hsc Cells Transduced With The Lentiviral Vector Containing A Shrna Targeting Bcl11A (this asset) | David Williams | P2 | CCC · 52 |
| Glutamine | Ain Shams University | P4 | BBB |
| Hydroxyurea | St. Jude Children's Research Hospital | P4 | BB |
| Nivestim | St. Jude Children's Research Hospital | P4 | BB |
| Crizanlizumab | Novartis Pharmaceuticals | P4 | BB |
| Magnesium Metallicum | Medical College of Wisconsin | P3 | BB |
| Fentanyl | University College Dublin | P4 | BB |
| Nepenthe | Oman Medical Speciality Board | P4 | BB |
| ARG | Tanta University | P3 | BB |
+42 more in the Sickle Cell Disease 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. Autologous Cd34+ Hsc Cells Transduced With The Lentiviral Vector Containing A Shrna Targeting Bcl11A in Sickle Cell Disease. 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-hsc-cells-transduced-with-the-lentiviral-vector-containing-a-shrna-targeting-bcl11a-sickle-cell-disease
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