Prednisone

Disease Of Genetic Or Genomic Mechanism

Instituto do Cancer do Estado de São PauloSmall moleculePhase 2Active

57/ 100

Provisional rating · Methodology v0.2

Rating breakdown

12 Active assets in the Disease Of Genetic Or Genomic Mechanism cohort.

Asset / Sponsor	Phase	Rating
Guanfacine Maimonides Medical Center	Phase 4	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
Ataluren PTC Therapeutics	Phase 3	B
Deramiocel Capricor Inc.	Phase 3	B
Human Allogeneic Cardiosphere-Derived Cells Capricor Inc.	Phase 2	B