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
Hemoglobinopathy
Rating breakdown
- Clinical Evidence
- 61/ 100
- Competitive Position
- 81/ 100
Competitive context
25 Active assets in the Hemoglobinopathy cohort.
| Asset / Sponsor | Phase | Rating |
|---|---|---|
Hydroxyurea National Institute of Blood and Marrow Transplant (NIBMT), Pakistan | Phase 3 | BB |
Deferasirox Novartis | Phase 4 | BB |
Fludarabine Masonic Cancer Center, University of Minnesota | Phase 2 | B |
PEGYINTERFERON-ALFA-2A Azienda Ospedaliera V. Cervello | Phase 4 | B |
Ribavirin Azienda Ospedaliera V. Cervello | Phase 4 | B |
View all 40 →
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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