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
Hematologic Disorder
Rating breakdown
- Clinical Evidence
- 61/ 100
- Competitive Position
- 89/ 100
Competitive context
31 Active assets in the Hematologic Disorder cohort.
| Asset / Sponsor | Phase | Rating |
|---|---|---|
Cyclophosphamide Baptist Health South Florida | Phase 2/3 | BBB |
Fludarabine Baptist Health South Florida | Phase 2 | BB |
Busulfanum Baptist Health South Florida | Phase 2 | BB |
Kogenate Bayer | Phase 4 | BB |
Hydroxyurea St. Jude Children's Research Hospital | Phase 3 | BB |
View all 50 →
Same molecule, other indications
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 is also rated in:
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