Autologous Cd34+ Cells Isolated From Mobilised Peripheral Blood By Positive Selection, Modified By Crispr/Cas9 (Clustered Regularly Interspaced Short Palindromic Repeats/Crispr-Associated Protein 9) Mediated Gene Editing Consisting Of A Guide Rna (Grna) Introduced Transiently As Ribonucleoprotein (Rnp) Complex, Targeting The Erythroid Lineage-Specific Enhancer Region Of Bcl11A (B-Cell Lymphoma/Leukemia 11A). The Site-Specific Cleavage By Cas9 Forms A Double Strand Break (Dsb), Which Is Subsequently Repaired By Nonhomologous End-Joining (Nhej), Leading To The Transcriptional Repression Of Bcl11A, A Repressor Of ?-Globin Gene Transcription
Thalassemia
Explore 22 assets in Thalassemia →Floatz Rating
BB67/100
Confidence
Indicative
Sponsor
Vertex Pharmaceuticals Incorporated
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.
56Moderate confidence
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT05477563CT.gov Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease | — | Recruiting | — | — | — |
NCT05356195CT.gov Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT) | — | Active Not Recruiting | — | — | — |
NCT04208529CT.gov A Long-term Follow-up Study in Participants Who Received CTX001 | — | Enrolling By Invitation | — | — | — |
NCT03655678CT.gov A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia | — | Completed | — | — | — |
Competitive Position
Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
89High confidence
Detailed axis rationale is planned and will be published soon.
Same indication · Thalassemia
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Autologous Cd34+ Cells Isolated From Mobilised Peripheral Blood By Positive Selection, Modified By Crispr/Cas9 (Clustered Regularly Interspaced Short Palindromic Repeats/Crispr-Associated Protein 9) Mediated Gene Editing Consisting Of A Guide Rna (Grna) Introduced Transiently As Ribonucleoprotein (Rnp) Complex, Targeting The Erythroid Lineage-Specific Enhancer Region Of Bcl11A (B-Cell Lymphoma/Leukemia 11A). The Site-Specific Cleavage By Cas9 Forms A Double Strand Break (Dsb), Which Is Subsequently Repaired By Nonhomologous End-Joining (Nhej), Leading To The Transcriptional Repression Of Bcl11A, A Repressor Of ?-Globin Gene Transcription (this asset) | Vertex Pharmaceuticals Incorporated | P3 | BB · 67 |
| Deferasirox | Novartis Pharmaceuticals | P4 | BBB |
| Hydroxyurea | Riphah International University | P4 | BB |
| Fludarabine | Memorial Sloan Kettering Cancer Center | P2/3 | BB |
| Zinc Sulfate | Fakultas Kedokteran Universitas Indonesia | P4 | BB |
| Busulfanum | University of California, San Francisco | P2/3 | BB |
| Ppsv23 | Fakultas Kedokteran Universitas Indonesia | P4 | BB |
| Etavopivat | Novo Nordisk A/S | P3 | BB |
| Conjugated Pneumococcal Vaccine | Fakultas Kedokteran Universitas Indonesia | P4 | BB |
+42 more in the Thalassemia cohort
Other indications for Autologous Cd34+ Cells Isolated From Mobilised Peripheral Blood By Positive Selection, Modified By Crispr/Cas9 (Clustered Regularly Interspaced Short Palindromic Repeats/Crispr-Associated Protein 9) Mediated Gene Editing Consisting Of A Guide Rna (Grna) Introduced Transiently As Ribonucleoprotein (Rnp) Complex, Targeting The Erythroid Lineage-Specific Enhancer Region Of Bcl11A (B-Cell Lymphoma/Leukemia 11A). The Site-Specific Cleavage By Cas9 Forms A Double Strand Break (Dsb), Which Is Subsequently Repaired By Nonhomologous End-Joining (Nhej), Leading To The Transcriptional Repression Of Bcl11A, A Repressor Of ?-Globin Gene Transcription
| Indication | Sponsor | Phase | Rating |
|---|---|---|---|
| Hematologic Disorder | — | P3 | BB · 70 |
| Disease Of Genetic Or Genomic Mechanism | — | P3 | BB · 69 |
| Hemoglobinopathy | — | P3 | BB · 68 |
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 Isolated From Mobilised Peripheral Blood By Positive Selection, Modified By Crispr/Cas9 (Clustered Regularly Interspaced Short Palindromic Repeats/Crispr-Associated Protein 9) Mediated Gene Editing Consisting Of A Guide Rna (Grna) Introduced Transiently As Ribonucleoprotein (Rnp) Complex, Targeting The Erythroid Lineage-Specific Enhancer Region Of Bcl11A (B-Cell Lymphoma/Leukemia 11A). The Site-Specific Cleavage By Cas9 Forms A Double Strand Break (Dsb), Which Is Subsequently Repaired By Nonhomologous End-Joining (Nhej), Leading To The Transcriptional Repression Of Bcl11A, A Repressor Of ?-Globin Gene Transcription in 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-isolated-from-mobilised-peripheral-blood-by-positive-selection-modified-by-crisprcas9-clustered-regularly-interspaced-short-palindromic-repeatscrispr-associated-protein-9-mediated-gene-editing-consisting-of-a-guide-rna-grna-introduced-transiently-as-ribonucleoprotein-rnp-complex-targeting-the-erythroid-lineage-specific-enhancer-region-of-bcl11a-b-cell-lymphomaleukemia-11a-the-site-specific-cleavage-by-cas9-forms-a-double-strand-break-dsb-which-is-subsequently-repaired-by-nonhomologous-end-joining-nhej-leading-to-the-transcriptional-repression-of-bcl11a-a-repressor-of-globin-gene-transcription-thalassemia
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