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Deferitrin

Beta-Thalassemia

Genzyme, a Sanofi CompanySmall moleculePhase 1/2Dormant
CCC
48/ 100
Provisional rating · Methodology v0.2

Rating breakdown

Clinical Evidence
37/ 100
Competitive Position
81/ 100

Competitive context

35 Active assets in the Beta-Thalassemia cohort.

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

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