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
Thalassemia
Rating breakdown
- Clinical Evidence
- 56/ 100
- Competitive Position
- 89/ 100
Competitive context
21 Active assets in the Thalassemia cohort.
| Asset / Sponsor | Phase | Rating |
|---|---|---|
Deferasirox Novartis Pharmaceuticals | Phase 4 | BBB |
Fludarabine Memorial Sloan Kettering Cancer Center | Phase 2/3 | BB |
Hydroxyurea Riphah International University | Phase 4 | BB |
ZINC SULFATE Fakultas Kedokteran Universitas Indonesia | Phase 4 | BB |
Busulphan University of California, San Francisco | Phase 2/3 | BB |
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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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