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.
Hemoglobinopathy
Explore 26 assets in Hemoglobinopathy →Floatz Rating
CC45/100
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 | — | — | — |
NCT04853576CT.gov A Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY) | — | Active Not Recruiting | — | — | — |
Competitive Position
Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
81High confidence
Detailed axis rationale is planned and will be published soon.
Same indication · Hemoglobinopathy
| 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 | CC · 45 |
| 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 | Vertex Pharmaceuticals Incorporated | P3 | BB |
| Hydroxyurea | National Institute of Blood and Marrow Transplant (NIBMT), Pakistan | P3 | BB |
| Deferasirox | Novartis | P4 | BB |
| Fludarabine | Masonic Cancer Center, University of Minnesota | P2 | B |
| Pegyinterferon-Alfa-2A | Azienda Ospedaliera V. Cervello | P4 | B |
| Ribavirin | Azienda Ospedaliera V. Cervello | P4 | B |
| Busulfanum | Beam Therapeutics Inc. | P2 | B |
| Thiotepa | University of California, San Francisco | P2 | B |
+32 more in the Hemoglobinopathy 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 |
|---|---|---|---|
| Beta-Thalassemia Major | — | P1/2 | CCC · 48 |
| 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
- 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 TranscriptionVertex Pharmaceuticals IncorporatedBB
- HydroxyureaNational Institute of Blood and Marrow Transplant (NIBMT), PakistanBB
- DeferasiroxNovartisBB
- FludarabineMasonic Cancer Center, University of MinnesotaB
- Pegyinterferon-Alfa-2AAzienda Ospedaliera V. CervelloB
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 Hemoglobinopathy. 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-hemoglobinopathy
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