Busulfanum

Hematologic Disorder

Baptist Health South FloridaSmall moleculePhase 2Active

69/ 100

Provisional rating · Methodology v0.2

Rating breakdown

31 Active assets in the Hematologic Disorder cohort.

Asset / Sponsor	Phase	Rating
Cyclophosphamide Baptist Health South Florida	Phase 2/3	BBB
Fludarabine Baptist Health South Florida	Phase 2	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 Vertex Pharmaceuticals Incorporated	Phase 3	BB
Kogenate Bayer	Phase 4	BB
Hydroxyurea St. Jude Children's Research Hospital	Phase 3	BB