Penicillin

Hemoglobinopathies

National Heart, Lung, and Blood Institute (NHLBI)Phase 3Dormant

60/ 100

Provisional rating · Methodology v0.2

Rating breakdown

26 Active assets in the Hemoglobinopathies cohort.

Asset / Sponsor	Phase	Rating
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 Vertex Pharmaceuticals Incorporated	Phase 3	BB
Hydroxyurea National Institute of Blood and Marrow Transplant (NIBMT), Pakistan	Phase 3	BB
Deferasirox Novartis	Phase 4	BB
Fludarabine Masonic Cancer Center, University of Minnesota	Phase 2	B
Busulphan Beam Therapeutics Inc.	Phase 2	B