22 assets in Thalassemia

Therapeutic assets addressing Thalassemia, rated under Floatz methodology v0.2 and sorted by current rating.

Asset	Sponsor	Phase	Rating
Fludarabine Thalassemia	Memorial Sloan Kettering Cancer Center	P2/3	BB (70)
Hydroxyurea Thalassemia	Riphah International University	P4	BB (70)
Etavopivat Thalassemia	Novo Nordisk A/S	P3	BB (68)
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 Thalassemia	Vertex Pharmaceuticals Incorporated	P3	BB (67)
Cyclophosphamide Thalassemia	Memorial Sloan Kettering Cancer Center	P2/3	BB (66)
Alemtuzumab Thalassemia	National Heart, Lung, and Blood Institute (NHLBI)	P2	BB (65)
Tacrolimus Thalassemia	Memorial Sloan Kettering Cancer Center	P2	BB (65)
Nivestim Thalassemia	St. Jude Children's Research Hospital	P2/3	BB (64)
Prednisone Thalassemia	Memorial Sloan Kettering Cancer Center	P2	BB (64)
Ruxolitinib Thalassemia	Novartis Pharmaceuticals	P4	B (62)
Panobinostat Thalassemia	Novartis Pharmaceuticals	P4	B (61)
Pentostatin Thalassemia	City of Hope Medical Center	P2	B (60)
Anti-Human T-Lymphocyte Immunoglobulin, Rabbit Thalassemia	Memorial Sloan Kettering Cancer Center	P2	CCC (53)
Bortezomib Thalassemia	Memorial Sloan Kettering Cancer Center	P2	CCC (53)
Dexasone Thalassemia	Memorial Sloan Kettering Cancer Center	P2	CCC (53)
Rituximab Thalassemia	Memorial Sloan Kettering Cancer Center	P2	CCC (53)
Sirolimus Thalassemia	National Heart, Lung, and Blood Institute (NHLBI)	P2	CCC (53)
Moxifloxacin Thalassemia	Novo Nordisk A/S	P1	CC (46)
Rabbit Anti-Thymocyte Globulin Thalassemia	City of Hope Medical Center	P1	CC (43)
Biotinylated Human Red Blood Cell Thalassemia	National Heart, Lung, and Blood Institute (NHLBI)	Early P1	C (39)
T-Cell Surface Antigen T4/Leu-3 Thalassemia	City of Hope Medical Center	P1	D (30)
Thalidomide Thalassemia	Riphah International University	N/A	D (30)