Therapeutic Asset Ratings

122 results matching 1 filter

133,990 assets · 8,802 sponsors · 3,128 indications

Methodology v0.2

Asset	Sponsor	Phase	Rating
Hydroxyurea Sickle Cell Disease	St. Jude Children's Research Hospital	P4	BB (71)
Crizanlizumab Sickle Cell Disease	Novartis Pharmaceuticals	P4	BB (70)
FILGRASTIM Sickle Cell Disease	St. Jude Children's Research Hospital	P4	BB (70)
NEPENTHE Sickle Cell Disease	Oman Medical Speciality Board	P4	BB (69)
ARG Sickle Cell Disease	Tanta University	P3	BB (69)
Deferasirox Sickle Cell Disease	Inova Health Care Services	P4	BB (67)
Cyclophosphamide Sickle Cell Disease	St. Jude Children's Research Hospital	P2/3	BB (67)
Busulphan Sickle Cell Disease	St. Jude Children's Research Hospital	P2/3	BB (66)
Mitapivat Sickle Cell Disease	Agios Pharmaceuticals, Inc.	P3	BB (65)
TACROLIMUS Sickle Cell Disease	Masonic Cancer Center, University of Minnesota	P2	BB (65)
DESFERRIOXAMINE B Sickle Cell Disease	Inova Health Care Services	P4	BB (64)
Acetylsalicylic Acid Sickle Cell Disease	Instituto Nacional de Investigacao em Saude, Angola	P3	B (63)
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 Sickle Cell Disease	Vertex Pharmaceuticals Incorporated	P3	B (63)
Fludarabine Sickle Cell Disease	St. Jude Children's Research Hospital	P3	B (63)
METHOTREXATE Sickle Cell Disease	City of Hope Medical Center	P2	B (63)
VITAMIN D Sickle Cell Disease	University of California, Davis	P2/3	B (63)
Evomela Sickle Cell Disease	Emory University	P3	B (62)
CYCLOSPORIN A Sickle Cell Disease	City of Hope Medical Center	P2	B (62)
Monomethyl Fumarate Sickle Cell Disease	Masonic Cancer Center, University of Minnesota	P2	B (62)
Romidepsin Sickle Cell Disease	St. Jude Children's Research Hospital	P2	B (62)
(±)-2-(O-CHLOROPHENYL)-2-(METHYLAMINO)CYCLOHEXANONE Sickle Cell Disease	University of Mississippi Medical Center	P4	B (61)
IMMUNOGLOBULIN G1 (HUMAN-RAT MONOCLONAL CAMPATH-1H .GAMMA.1-CHAIN ANTI-HUMAN ANTIGEN CD52), DISULFIDE WITH HUMAN-RAT MONOCLONAL CAMPATH-1H LIGHT CHAIN, DIMER Sickle Cell Disease	St. Jude Children's Research Hospital	P3	B (61)
PREDNISONE TABLETS Sickle Cell Disease	Memorial Sloan Kettering Cancer Center	P3	B (61)
Thiotepa Sickle Cell Disease	St. Jude Children's Research Hospital	P2	B (61)
Deferiprone Sickle Cell Disease	Inova Health Care Services	P4	B (60)

Showing 1 to 25 of 122 assets

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