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
Transfusion Dependent Beta-Thalassaemia
Rating breakdown
- Clinical Evidence
- 28/ 100
- Competitive Position
- 95/ 100
Competitive context
31 Active assets in the Transfusion Dependent Beta-Thalassaemia cohort.
| Asset / Sponsor | Phase | Rating |
|---|---|---|
Deferasirox Pakistan Blood and Marrow Transplant (PBMT) Group | Phase 4 | BBB |
Deferiprone Pakistan Blood and Marrow Transplant (PBMT) Group | Phase 4 | BBB |
Hydroxyurea Bahria University | Phase 4 | BBB |
TACROLIMUS M.D. Anderson Cancer Center | Phase 4 | BBB |
DESFERRIOXAMINE B Pakistan Blood and Marrow Transplant (PBMT) Group | Phase 4 | BBB |
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:
Are you the sponsor?
Submit your data room for a Verified Rating. The public-data rating remains visible alongside.
Contact →