Autologous Cd34+ Cells Transduced With Tns9.3.55

Beta Thalassemia

Daniel BauerunclassifiedPhase 1/2Active

CCC

50/ 100

Provisional rating · Methodology v0.2

Rating breakdown

34 Active assets in the Beta Thalassemia cohort.

Asset / Sponsor	Phase	Rating
Deferasirox Kevin H.M. Kuo, MD, MSc, FRCPC	Phase 4	BBB
Desferrioxamine B Ain Shams University	Phase 4	BBB
Hydroxyurea National Heart, Lung, and Blood Institute (NHLBI)	Phase 2/3	BB
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 Daniel Bauer	Phase 3	BB
Thalidomide National Institute of Blood and Marrow Transplant (NIBMT), Pakistan	Phase 2/3	BB