Autologous Human Cd34+ Hematopoietic Stem/Precursor Cells (Hspcs), Obtained From Peripheral Blood By Leukapheresis From Plerixaformobilized Sickle Cell Disease (Scd) Patients Or Granulocyte Colony Stimulating Factor (G-Csf) And Plerixafor-Mobilized Transfusion Dependent Betathalassemia (Tdt) Patients, Genetically Modified Ex Vivo By Crispr/Cas12A (Clustered Regularly Interspaced Palindromic Repeats/Modified Acidaminococcus Sp. Cas12A) Endonuclease Complexed With A Guide Rna (Grna) That Targets The Ccaat-Box Region Of Both Gamma Globin Gene (Hbg1 And Hgb2) Promoters On Chromosome 11, Creating Indels That Disrupt Repressor Binding And Increase Gamma Globin Expression. The Editing Components Are Introduced Into The Target Cell Population As A Ribonucleoprotein Complex By Electroporation. The Cell Suspension Is Enriched For Cd34+ Cells Using Magnetic Bead Separation. Following Electroporation, The Cells Are Cultured In Media Containing Thrombopoietin, Fms-Related Tyrosine Kinase 3 Ligand (Flt3L), And Stem Cell Factor (Scf). The Substance Consists Of Cells With ?70% Cd34/Cd45+ Purity And ?70% On-Target Editing.
Beta-Thalassemia Major
Explore 31 assets in Beta-Thalassemia Major →Floatz Rating
CCC48/100
Confidence
Indicative
Sponsor
Editas Medicine, Inc.
Modality
Cell therapy
Development Phase
Phase 1/2
Status
Active
Evidence ledger · v0.2
Clinical Evidence
Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
33Moderate confidence
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT05444894CT.gov EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT) | — | Active Not Recruiting | — | — | — |
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 Human Cd34+ Hematopoietic Stem/Precursor Cells (Hspcs), Obtained From Peripheral Blood By Leukapheresis From Plerixaformobilized Sickle Cell Disease (Scd) Patients Or Granulocyte Colony Stimulating Factor (G-Csf) And Plerixafor-Mobilized Transfusion Dependent Betathalassemia (Tdt) Patients, Genetically Modified Ex Vivo By Crispr/Cas12A (Clustered Regularly Interspaced Palindromic Repeats/Modified Acidaminococcus Sp. Cas12A) Endonuclease Complexed With A Guide Rna (Grna) That Targets The Ccaat-Box Region Of Both Gamma Globin Gene (Hbg1 And Hgb2) Promoters On Chromosome 11, Creating Indels That Disrupt Repressor Binding And Increase Gamma Globin Expression. The Editing Components Are Introduced Into The Target Cell Population As A Ribonucleoprotein Complex By Electroporation. The Cell Suspension Is Enriched For Cd34+ Cells Using Magnetic Bead Separation. Following Electroporation, The Cells Are Cultured In Media Containing Thrombopoietin, Fms-Related Tyrosine Kinase 3 Ligand (Flt3L), And Stem Cell Factor (Scf). The Substance Consists Of Cells With ?70% Cd34/Cd45+ Purity And ?70% On-Target Editing. (this asset) | Editas Medicine, Inc. | P1/2 | CCC · 48 |
| 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 Human Cd34+ Hematopoietic Stem/Precursor Cells (Hspcs), Obtained From Peripheral Blood By Leukapheresis From Plerixaformobilized Sickle Cell Disease (Scd) Patients Or Granulocyte Colony Stimulating Factor (G-Csf) And Plerixafor-Mobilized Transfusion Dependent Betathalassemia (Tdt) Patients, Genetically Modified Ex Vivo By Crispr/Cas12A (Clustered Regularly Interspaced Palindromic Repeats/Modified Acidaminococcus Sp. Cas12A) Endonuclease Complexed With A Guide Rna (Grna) That Targets The Ccaat-Box Region Of Both Gamma Globin Gene (Hbg1 And Hgb2) Promoters On Chromosome 11, Creating Indels That Disrupt Repressor Binding And Increase Gamma Globin Expression. The Editing Components Are Introduced Into The Target Cell Population As A Ribonucleoprotein Complex By Electroporation. The Cell Suspension Is Enriched For Cd34+ Cells Using Magnetic Bead Separation. Following Electroporation, The Cells Are Cultured In Media Containing Thrombopoietin, Fms-Related Tyrosine Kinase 3 Ligand (Flt3L), And Stem Cell Factor (Scf). The Substance Consists Of Cells With ?70% Cd34/Cd45+ Purity And ?70% On-Target Editing.
| Indication | Sponsor | Phase | Rating |
|---|---|---|---|
| Hemoglobinopathy | — | P1/2 | CC · 45 |
| Sickle Cell Disease | — | P1/2 | 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 Human Cd34+ Hematopoietic Stem/Precursor Cells (Hspcs), Obtained From Peripheral Blood By Leukapheresis From Plerixaformobilized Sickle Cell Disease (Scd) Patients Or Granulocyte Colony Stimulating Factor (G-Csf) And Plerixafor-Mobilized Transfusion Dependent Betathalassemia (Tdt) Patients, Genetically Modified Ex Vivo By Crispr/Cas12A (Clustered Regularly Interspaced Palindromic Repeats/Modified Acidaminococcus Sp. Cas12A) Endonuclease Complexed With A Guide Rna (Grna) That Targets The Ccaat-Box Region Of Both Gamma Globin Gene (Hbg1 And Hgb2) Promoters On Chromosome 11, Creating Indels That Disrupt Repressor Binding And Increase Gamma Globin Expression. The Editing Components Are Introduced Into The Target Cell Population As A Ribonucleoprotein Complex By Electroporation. The Cell Suspension Is Enriched For Cd34+ Cells Using Magnetic Bead Separation. Following Electroporation, The Cells Are Cultured In Media Containing Thrombopoietin, Fms-Related Tyrosine Kinase 3 Ligand (Flt3L), And Stem Cell Factor (Scf). The Substance Consists Of Cells With ?70% Cd34/Cd45+ Purity And ?70% On-Target Editing. 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-human-cd34-hematopoietic-stemprecursor-cells-hspcs-obtained-from-peripheral-blood-by-leukapheresis-from-plerixaformobilized-sickle-cell-disease-scd-patients-or-granulocyte-colony-stimulating-factor-g-csf-and-plerixafor-mobilized-transfusion-dependent-betathalassemia-tdt-patients-genetically-modified-ex-vivo-by-crisprcas12a-clustered-regularly-interspaced-palindromic-repeatsmodified-acidaminococcus-sp-cas12a-endonuclease-complexed-with-a-guide-rna-grna-that-targets-the-ccaat-box-region-of-both-gamma-globin-gene-hbg1-and-hgb2-promoters-on-chromosome-11-creating-indels-that-disrupt-repressor-binding-and-increase-gamma-globin-expression-the-editing-components-are-introduced-into-the-target-cell-population-as-a-ribonucleoprotein-complex-by-electroporation-the-cell-suspension-is-enriched-for-cd34-cells-using-magnetic-bead-separation-following-electroporation-the-cells-are-cultured-in-media-containing-thrombopoietin-fms-related-tyrosine-kinase-3-ligand-flt3l-and-stem-cell-factor-scf-the-substance-consists-of-cells-with-70-cd34cd45-purity-and-70-on-target-editing-beta-thalassemia-major
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