.GAMMA.-CYCLODEXTRIN, 6A,6B,6C,6D,6E,7F,6G,6H-OCTAKIS-S-(2-CARBOXYETHYL)-6A,6B,6C,6D,6E,7F,6G,6H-OCTATHIO-

Beta-Thalassemia

Federal Research Institute of Pediatric Hematology, Oncology and ImmunologySmall moleculePhase 2/3Dormant

CCC

55/ 100

Provisional rating · Methodology v0.2

Rating breakdown

35 Active assets in the Beta-Thalassemia cohort.

Asset / Sponsor	Phase	Rating
Deferasirox Kevin H.M. Kuo, MD, MSc, FRCPC	Phase 4	BBB
DESFERRIOXAMINE B Ain Shams University	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