31 assets in Beta-Thalassemia Major

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

Asset	Sponsor	Phase	Rating
Deferasirox Beta-Thalassemia Major	Pakistan Blood and Marrow Transplant (PBMT) Group	P4	BBB (79)
Deferiprone Beta-Thalassemia Major	Pakistan Blood and Marrow Transplant (PBMT) Group	P4	BBB (77)
Hydroxyurea Beta-Thalassemia Major	Bahria University	P4	BBB (77)
Desferrioxamine B Beta-Thalassemia Major	Pakistan Blood and Marrow Transplant (PBMT) Group	P4	BBB (75)
Busulfanum Beta-Thalassemia Major	M.D. Anderson Cancer Center	P4	BBB (73)
Fludarabine Beta-Thalassemia Major	M.D. Anderson Cancer Center	P4	BBB (73)
Cyclophosphamide Beta-Thalassemia Major	M.D. Anderson Cancer Center	P4	BB (71)
Metformin Beta-Thalassemia Major	Bahria University	P4	BB (68)
Thalidomide Beta-Thalassemia Major	National Institute of Blood and Marrow Transplant (NIBMT), Pakistan	P3	BB (67)
Luspatercept Beta-Thalassemia Major	Bristol-Myers Squibb	P4	BB (65)
cs-101 Beta-Thalassemia Major	CorrectSequence Therapeutics Co., Ltd	P2	B (60)
Mitapivat Beta-Thalassemia Major	Agios Pharmaceuticals, Inc.	P3	B (60)
Dulaglutide Beta-Thalassemia Major	Ain Shams University	P2	B (57)
Autologous Cd34+ Cells Transduced With Tns9.3.55 Beta-Thalassemia Major	Daniel Bauer	P1/2	CCC (54)
Rituximab Beta-Thalassemia Major	M.D. Anderson Cancer Center	P1/2	CCC (54)
Alemtuzumab Beta-Thalassemia Major	Paul Szabolcs	P1/2	CCC (52)
Autologous Human Cd34+ Hematopoietic Stem/Precursor Cells (Hspcs), Obtained From Peripheral Blood By Leukapheresis From Plerixaformobilized Sickle Cell Disease (Scd) Patients Or Granulocyte Colony Stimulating Factor (G-Csf) And Plerixafor-Mobilized Transfusion Dependent Betathalassemia (Tdt) Patients, Genetically Modified Ex Vivo By Crispr/Cas12A (Clustered Regularly Interspaced Palindromic Repeats/Modified Acidaminococcus Sp. Cas12A) Endonuclease Complexed With A Guide Rna (Grna) That Targets The Ccaat-Box Region Of Both Gamma Globin Gene (Hbg1 And Hgb2) Promoters On Chromosome 11, Creating Indels That Disrupt Repressor Binding And Increase Gamma Globin Expression. The Editing Components Are Introduced Into The Target Cell Population As A Ribonucleoprotein Complex By Electroporation. The Cell Suspension Is Enriched For Cd34+ Cells Using Magnetic Bead Separation. Following Electroporation, The Cells Are Cultured In Media Containing Thrombopoietin, Fms-Related Tyrosine Kinase 3 Ligand (Flt3L), And Stem Cell Factor (Scf). The Substance Consists Of Cells With ?70% Cd34/Cd45+ Purity And ?70% On-Target Editing. Beta-Thalassemia Major	Editas Medicine, Inc.	P1/2	CCC (48)
cd45ra depleted leukocytes Beta-Thalassemia Major	Paul Szabolcs	P1/2	CCC (48)
kl003 cell Beta-Thalassemia Major	Kanglin Biotechnology (Hangzhou) Co., Ltd.	P1/2	CCC (48)
Thiotepa Beta-Thalassemia Major	Paul Szabolcs	P1/2	CCC (48)
Β-Globin Restored Autologous Hsc Beta-Thalassemia Major	Bioray Laboratories	P1/2	CCC (48)
Acetylcysteine Beta-Thalassemia Major	Assiut University	P1	CC (46)
Petadeferitrin Beta-Thalassemia Major	Pharmacosmos A/S	P2	CC (46)
Autologous Bone Marrow Derived Cd34+ Hspcs Electroporated With Bcl11A Enhancer Targeting Cas9 Ribonucleoprotein Beta-Thalassemia Major	Daniel Bauer	P1	CC (43)
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 Major	Daniel Bauer	P1	CC (43)
Gmcn-508B Beta-Thalassemia Major	First Affiliated Hospital of Guangxi Medical University	Early P1	C (39)
Yolt-204 Beta-Thalassemia Major	First Affiliated Hospital of Guangxi Medical University	Early P1	C (39)
et-01 Beta-Thalassemia Major	EdiGene (GuangZhou) Inc.	P1	C (36)
bd211 Beta-Thalassemia Major	Shanghai BDgene Co., Ltd.	N/A	D (30)
Vgb-Ex01 Beta-Thalassemia Major	Institute of Hematology & Blood Diseases Hospital, China	N/A	D (30)
Γ-Globin Reactivated Autologous Hematopoietic Stem Cells Beta-Thalassemia Major	Bioray Laboratories	N/A	D (30)