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
Hemoglobinopathies
Rating breakdown
- Clinical Evidence
- 61/ 100
- Competitive Position
- 81/ 100
Competitive context
25 Active assets in the Hemoglobinopathies 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 |
Pegylated Interferon Alpha2A Azienda Ospedaliera V. Cervello | Phase 4 | B |
Busulphan Beam Therapeutics Inc. | Phase 2 | B |
View all 41 →
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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