Hydroxyurea

Hemoglobinopathy

National Institute of Blood and Marrow Transplant (NIBMT), PakistanSmall moleculePhase 3Active

66/ 100

Provisional rating · Methodology v0.2

Rating breakdown

25 Active assets in the Hemoglobinopathy 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
Deferasirox Novartis	Phase 4	BB
Fludarabine Masonic Cancer Center, University of Minnesota	Phase 2	B
Pegyinterferon-Alfa-2A Azienda Ospedaliera V. Cervello	Phase 4	B
Busulfanum Beam Therapeutics Inc.	Phase 2	B