Autologous Bone Marrow Derived Cd34+ Hspcs Electroporated With Bcl11A Enhancer Targeting Cas9 Ribonucleoprotein
Sickle Cell Disease
Explore 133 assets in Sickle Cell Disease →Floatz Rating
C37/100
Confidence
Indicative
Sponsor
Daniel Bauer
Modality
gene_therapy
Development Phase
Phase 1
Status
Active
Evidence ledger · v0.2
Clinical Evidence
Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
28Indicative
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT06647979CT.gov Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies | — | Recruiting | — | — | — |
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 Bone Marrow Derived Cd34+ Hspcs Electroporated With Bcl11A Enhancer Targeting Cas9 Ribonucleoprotein (this asset) | Daniel Bauer | P1 | C · 37 |
| Glutamine | Ain Shams University | P4 | BBB |
| Hydroxyurea | St. Jude Children's Research Hospital | P4 | BB |
| Magnesium Metallicum | Medical College of Wisconsin | P3 | BB |
| Nivestim | St. Jude Children's Research Hospital | P4 | BB |
| Crizanlizumab | Novartis Pharmaceuticals | P4 | BB |
| Nepenthe | Oman Medical Speciality Board | P4 | BB |
| ARG | Tanta University | P3 | BB |
| Fentanyl | University College Dublin | P4 | BB |
+42 more in the Sickle Cell Disease cohort
Other indications for Autologous Bone Marrow Derived Cd34+ Hspcs Electroporated With Bcl11A Enhancer Targeting Cas9 Ribonucleoprotein
| Indication | Sponsor | Phase | Rating |
|---|---|---|---|
| Beta-Thalassemia Major | — | P1 | CC · 43 |
| Beta Thalassemia | — | P1 | CC · 41 |
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 Bone Marrow Derived Cd34+ Hspcs Electroporated With Bcl11A Enhancer Targeting Cas9 Ribonucleoprotein 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-bone-marrow-derived-cd34-hspcs-electroporated-with-bcl11a-enhancer-targeting-cas9-ribonucleoprotein-sickle-cell-disease
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