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Beta Thalassemia

National Heart, Lung, and Blood Institute (NHLBI)Small moleculePhase 1/2Active

47/ 100

Provisional rating · Methodology v0.2

Rating breakdown

34 Active assets in the Beta Thalassemia cohort.

Asset / Sponsor	Phase	Rating
DESFERRIOXAMINE B Ain Shams University	Phase 4	BBB
Deferasirox Kevin H.M. Kuo, MD, MSc, FRCPC	Phase 4	BBB
Hydroxyurea National Heart, Lung, and Blood Institute (NHLBI)	Phase 2/3	BB
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 Daniel Bauer	Phase 3	BB
Thalidomide National Institute of Blood and Marrow Transplant (NIBMT), Pakistan	Phase 2/3	BB