35 assets in Beta Thalassemia

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

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 Beta Thalassemia	Daniel Bauer	P3	BB (68)
Hydroxyurea Beta Thalassemia	National Heart, Lung, and Blood Institute (NHLBI)	P2/3	BB (68)
Thalidomide Beta Thalassemia	National Institute of Blood and Marrow Transplant (NIBMT), Pakistan	P2/3	BB (66)
Fludarabine Beta Thalassemia	Memorial Sloan Kettering Cancer Center	P2	BB (64)
Luspatercept Beta Thalassemia	Bristol-Myers Squibb	P3	BB (64)
Busulfanum Beta Thalassemia	New York Medical College	P2	B (61)
Acetylcysteine Beta Thalassemia	Tanta University	P3	B (60)
Autologous Cd34+ Cells Encoding .Beta.A-T87Q-Globin Gene Beta Thalassemia	CorrectSequence Therapeutics Co., Ltd	P3	B (60)
D-Thioctic Acid Beta Thalassemia	Tanta University	P3	B (60)
L-Carnitine Beta Thalassemia	Tanta University	P3	B (60)
Sirolimus Beta Thalassemia	National Heart, Lung, and Blood Institute (NHLBI)	P2	B (60)
Alemtuzumab Beta Thalassemia	National Heart, Lung, and Blood Institute (NHLBI)	P2	B (58)
Sildenafil Beta Thalassemia	Tanta University	P3	B (58)
Cyclophosphamide Beta Thalassemia	Memorial Sloan Kettering Cancer Center	P2	B (57)
Anti-Human T-Lymphocyte Immunoglobulin, Rabbit Beta Thalassemia	Memorial Sloan Kettering Cancer Center	P2	CCC (53)
Bortezomib Beta Thalassemia	Memorial Sloan Kettering Cancer Center	P2	CCC (53)
Dexasone Beta Thalassemia	Memorial Sloan Kettering Cancer Center	P2	CCC (53)
Rituximab Beta Thalassemia	Memorial Sloan Kettering Cancer Center	P2	CCC (53)
Tacrolimus Beta Thalassemia	Memorial Sloan Kettering Cancer Center	P2	CCC (53)
Vamifeport Beta Thalassemia	Vifor (International) Inc.	P2	CCC (53)
Autologous Cd34+ Cells Transduced With Tns9.3.55 Beta Thalassemia	Daniel Bauer	P1/2	CCC (50)
Briquilimab Beta Thalassemia	National Heart, Lung, and Blood Institute (NHLBI)	P1/2	CC (47)
Nivestim Beta Thalassemia	National Heart, Lung, and Blood Institute (NHLBI)	P1/2	CC (47)
Plerixafor Beta Thalassemia	National Heart, Lung, and Blood Institute (NHLBI)	P1/2	CC (47)
Abatacept Recombinant Beta Thalassemia	National Heart, Lung, and Blood Institute (NHLBI)	P1/2	CC (45)
als20 Beta Thalassemia	Children's Hospital of Philadelphia	P1/2	CC (45)
Brl-101 Beta Thalassemia	Bioray Laboratories	P1/2	CC (45)
cs-101 Beta Thalassemia	Children's Hospital of Fudan University	Early P1	CC (45)
Vebeglogene Autotemcel Beta Thalassemia	Lantu Biopharma	P1/2	CC (45)
bd211 Beta Thalassemia	Shanghai BDgene Co., Ltd.	P1	CC (43)
Fostamatinib Beta Thalassemia	National Heart, Lung, and Blood Institute (NHLBI)	P1	CC (43)
9mw3011 Beta Thalassemia	Mabwell (Shanghai) Bioscience Co., Ltd.	P1	CC (41)
Autologous Bone Marrow Derived Cd34+ Hspcs Electroporated With Bcl11A Enhancer Targeting Cas9 Ribonucleoprotein Beta Thalassemia	Daniel Bauer	P1	CC (41)
Β-Globin Restored Autologous Hematopoietic Stem Cells Beta Thalassemia	Shenzhen Hemogen	Early P1	C (39)
Vgb-Ex01 Beta Thalassemia	Institute of Hematology & Blood Diseases Hospital, China	N/A	D (30)