Solu-Cortef

Genetic Diseases, Inborn

University of MinnesotaPhase 1Dormant

47/ 100

Provisional rating · Methodology v0.2

Rating breakdown

13 Active assets in the Genetic Diseases, Inborn cohort.

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 Vertex Pharmaceuticals Incorporated	Phase 3	BB
Guanfacine Maimonides Medical Center	Phase 4	BB
Ataluren PTC Therapeutics	Phase 3	B
Deramiocel Capricor Inc.	Phase 3	B
PREDNISONE TABLETS National Cancer Institute (NCI)	Phase 2	B