Therapeutic Asset Ratings

122 results matching 1 filter

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

Methodology v0.2

Asset	Sponsor	Phase	Rating
VAMIFEPORT Sickle Cell Disease	Vifor (International) Inc.	P2	CCC (51)
Letrozole Sickle Cell Disease	Pfizer	P1	CCC (49)
ALENDRONIC ACID Sickle Cell Disease	University of California, Davis	P2	CCC (48)
AMMONIA N-13 Sickle Cell Disease	St. Jude Children's Research Hospital	P2	CCC (48)
Anumigilimab Sickle Cell Disease	CSL Behring	P2	CCC (48)
CIS-DIAMMINEDICHLOROPLATINUM II Sickle Cell Disease	Children's National Research Institute	P2	CCC (48)
DARATUMUMAB Sickle Cell Disease	Children's National Research Institute	P2	CCC (48)
Zinc Sickle Cell Disease	University of California, San Francisco	P2	CCC (48)
BIOTINYLATED HUMAN RED BLOOD CELL Sickle Cell Disease	National Heart, Lung, and Blood Institute (NHLBI)	P1	CC (45)
Inclacumab Sickle Cell Disease	Pfizer	P3	CC (44)
MIDAZOLAM Sickle Cell Disease	Pfizer	P1	CC (44)
AUTOLOGOUS CD34+ CELLS ENCODING .BETA.A-T87Q-GLOBIN GENE Sickle Cell Disease	Editas Medicine, Inc.	P1/2	CC (44)
ab1 Sickle Cell Disease	Nirmish Shah	P1/2	CC (41)
Autologous human CD34+ hematopoietic stem/precursor cells (HSPCs), obtained from peripheral blood by leukapheresis from plerixaformobilized sickle cell disease (SCD) patients or granulocyte colony stimulating factor (G-CSF) and plerixafor-mobilized transfusion dependent betathalassemia (TDT) patients, genetically modified ex vivo by CRISPR/Cas12a (clustered regularly interspaced palindromic repeats/modified Acidaminococcus sp. Cas12a) endonuclease complexed with a guide RNA (gRNA) that targets the CCAAT-box region of both gamma globin gene (HBG1 and HGB2) promoters on chromosome 11, creating indels that disrupt repressor binding and increase gamma globin expression. The editing components are introduced into the target cell population as a ribonucleoprotein complex by electroporation. The cell suspension is enriched for CD34+ cells using magnetic bead separation. Following electroporation, the cells are cultured in media containing thrombopoietin, Fms-related tyrosine kinase 3 ligand (Flt3L), and stem cell factor (SCF). The substance consists of cells with ?70% CD34/CD45+ purity and ?70% on-target editing. Sickle Cell Disease	Editas Medicine, Inc.	P1/2	CC (41)
Beam-101 Sickle Cell Disease	Beam Therapeutics Inc.	P1/2	CC (41)
Benserazide Sickle Cell Disease	Phoenicia BioScience	P1/2	CC (41)
Crispr_Scd001 Sickle Cell Disease	Mark Walters, MD	P1/2	CC (41)
Dream01 Drug Product Sickle Cell Disease	Assistance Publique - Hôpitaux de Paris	P1/2	CC (41)
itu512 Sickle Cell Disease	Novartis Pharmaceuticals	P1/2	CC (41)
nula-cel Sickle Cell Disease	Kamau Therapeutics	P1/2	CC (41)
Ruxolitinib Sickle Cell Disease	Arkansas Children's Hospital Research Institute	P1/2	CC (41)
bkt140 Sickle Cell Disease	St. Jude Children's Research Hospital	P1	CC (40)
FLURBIPROFEN Sickle Cell Disease	Pfizer	P1	CC (40)
FUROSEMIDE Sickle Cell Disease	Pfizer	P1	CC (40)
MOXIFLOXACIN Sickle Cell Disease	Novo Nordisk A/S	P1	CC (40)

Showing 76 to 100 of 122 assets

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