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
Genetic Diseases, Inborn
Rating breakdown
- Clinical Evidence
- 56/ 100
- Competitive Position
- 95/ 100
Competitive context
12 Active assets in the Genetic Diseases, Inborn cohort.
| Asset / Sponsor | Phase | Rating |
|---|---|---|
Guanfacine Maimonides Medical Center | Phase 4 | BB |
Ataluren PTC Therapeutics | Phase 3 | B |
Deramiocel Capricor Inc. | Phase 3 | B |
PREDNISONE TABLETS Instituto do Cancer do Estado de São Paulo | Phase 2 | B |
Allogeneic Cardiosphere-Derived Cells Capricor Inc. | Phase 2 | B |
View all 24 →
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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