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
Disease Of Genetic Or Genomic Mechanism
Rating breakdown
- Clinical Evidence
- 56/ 100
- Competitive Position
- 95/ 100
Competitive context
12 Active assets in the Disease Of Genetic Or Genomic Mechanism cohort.
| Asset / Sponsor | Phase | Rating |
|---|---|---|
Guanfacine Maimonides Medical Center | Phase 4 | BB |
Ataluren PTC Therapeutics | Phase 3 | B |
Deramiocel Capricor Inc. | Phase 3 | B |
Human Allogeneic Cardiosphere-Derived Cells Capricor Inc. | Phase 2 | B |
Prednisone Instituto do Cancer do Estado de São Paulo | Phase 2 | B |
View all 21 →
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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