26 assets in Hemoglobinopathy

Therapeutic assets addressing Hemoglobinopathy, 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 Hemoglobinopathy	Vertex Pharmaceuticals Incorporated	P3	BB (68)
Hydroxyurea Hemoglobinopathy	National Institute of Blood and Marrow Transplant (NIBMT), Pakistan	P3	BB (66)
Fludarabine Hemoglobinopathy	Masonic Cancer Center, University of Minnesota	P2	B (63)
Busulfanum Hemoglobinopathy	Beam Therapeutics Inc.	P2	B (62)
Thiotepa Hemoglobinopathy	University of California, San Francisco	P2	B (61)
Alemtuzumab Hemoglobinopathy	Masonic Cancer Center, University of Minnesota	P2	B (60)
Cyclophosphamide Hemoglobinopathy	Masonic Cancer Center, University of Minnesota	P2	B (58)
Cyclosporin A Hemoglobinopathy	Memorial Sloan Kettering Cancer Center	P2	B (58)
Tacrolimus Hemoglobinopathy	Masonic Cancer Center, University of Minnesota	P2	B (58)
Anti-Human T-Lymphocyte Immunoglobulin, Rabbit Hemoglobinopathy	Masonic Cancer Center, University of Minnesota	P2	CCC (53)
Clofarabine Hemoglobinopathy	Memorial Sloan Kettering Cancer Center	P2	CCC (53)
Hpc, Cord Blood Hemoglobinopathy	Memorial Sloan Kettering Cancer Center	P2	CCC (53)
Plerixafor Hemoglobinopathy	Masonic Cancer Center, University of Minnesota	P2	CCC (53)
Sirolimus Hemoglobinopathy	Masonic Cancer Center, University of Minnesota	P2	CCC (53)
Thalidomide Hemoglobinopathy	National Institute of Blood and Marrow Transplant (NIBMT), Pakistan	P2	CCC (53)
Rimiducid Hemoglobinopathy	Bellicum Pharmaceuticals	P1/2	CC (46)
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. Hemoglobinopathy	Editas Medicine, Inc.	P1/2	CC (45)
Melphalan Hemoglobinopathy	Washington University School of Medicine	P1/2	CC (45)
Bpx-501 T Cells Hemoglobinopathy	Bellicum Pharmaceuticals	P1/2	CC (44)
Pentostatin Hemoglobinopathy	City of Hope Medical Center	P1	CC (41)
Rabbit Anti-Thymocyte Globulin Hemoglobinopathy	City of Hope Medical Center	P1	CC (41)
Biotinylated Human Red Blood Cell Hemoglobinopathy	National Heart, Lung, and Blood Institute (NHLBI)	Early P1	C (39)
Levetiracetam Hemoglobinopathy	Masonic Cancer Center, University of Minnesota	N/A	D (30)
T-Cell Surface Antigen T4/Leu-3 Hemoglobinopathy	City of Hope Medical Center	P1	D (30)
Yolt-204 Hemoglobinopathy	Guangzhou Women and Children's Medical Center	Early P1	NR ()