133 assets in Sickle Cell Disease

Therapeutic assets addressing Sickle Cell Disease, rated under Floatz methodology v0.2 and sorted by current rating.

Asset	Sponsor	Phase	Rating
Hbi-002 Sickle Cell Disease	Hillhurst Biopharmaceuticals, Inc.	P1/2	CCC (55)
Metformin Sickle Cell Disease	Pfizer	P1	CCC (55)
Tocilizumab Sickle Cell Disease	Assistance Publique - Hôpitaux de Paris	P3	CCC (55)
Abatacept Recombinant Sickle Cell Disease	St. Jude Children's Research Hospital	P2	CCC (54)
Anti-Human T-Lymphocyte Immunoglobulin, Rabbit Sickle Cell Disease	Masonic Cancer Center, University of Minnesota	P2	CCC (54)
Clonidine Sickle Cell Disease	Centre Hospitalier Universitaire Saint Pierre	P4	CCC (54)
Mesna Sickle Cell Disease	Columbia University	P2	CCC (54)
Pentostatin Sickle Cell Disease	City of Hope Medical Center	P2	CCC (54)
Empagliflozin Sickle Cell Disease	University of Illinois at Chicago	P2	CCC (53)
Osivelotor Sickle Cell Disease	Pfizer	P2/3	CCC (53)
Tarperprumig Sickle Cell Disease	Alexion Pharmaceuticals, Inc.	P2	CCC (53)
Autologous Cd34+ Cells Transduced With Tns9.3.55 Sickle Cell Disease	Daniel Bauer	P2	CCC (52)
Autologous Cd34+ Hsc Cells Transduced With The Lentiviral Vector Containing A Shrna Targeting Bcl11A Sickle Cell Disease	David Williams	P2	CCC (52)
Azathioprine Sickle Cell Disease	St. Jude Children's Research Hospital	P2	CCC (52)
Bortezomib Sickle Cell Disease	Memorial Sloan Kettering Cancer Center	P2	CCC (52)
Hemopexin (Plasma Derived) Sickle Cell Disease	CSL Behring	P2	CCC (52)
Iloprost Sickle Cell Disease	University Hospital, Rouen	P3	CCC (52)
Lovotibeglogene Autotemcel Sickle Cell Disease	Genetix Biotherapeutics Inc.	P3	CCC (52)
pcv21 Sickle Cell Disease	Sanofi	P3	CCC (52)
Pociredir Sickle Cell Disease	Fulcrum Therapeutics	P2	CCC (52)
Prevnar 20 Sickle Cell Disease	Sanofi	P3	CCC (52)
Red Blood Cell Sickle Cell Disease	University of Pittsburgh	P3	CCC (52)
Regadenoson Sickle Cell Disease	St. Jude Children's Research Hospital	P2	CCC (52)
Rilzabrutinib Sickle Cell Disease	Sanofi	P3	CCC (52)
Tetrahydrouridine Sickle Cell Disease	Novo Nordisk A/S	P2	CCC (52)
Tovinontrine Sickle Cell Disease	Cardurion Pharmaceuticals, Inc.	P2	CCC (52)
Defibrotide Sickle Cell Disease	New York Medical College	P2	CCC (51)
Epeleuton Sickle Cell Disease	Afimmune	P2	CCC (51)
Rifaximin Sickle Cell Disease	Bausch Health Americas, Inc.	P2	CCC (51)
Vamifeport Sickle Cell Disease	Vifor (International) Inc.	P2	CCC (51)
Epoetin Alfa Sickle Cell Disease	Julia Xu	P1/2	CCC (50)
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)
Daratumumab Sickle Cell Disease	Children's National Research Institute	P2	CCC (48)
Zinc Sickle Cell Disease	University of California, San Francisco	P2	CCC (48)
Bms-986470 Sickle Cell Disease	Bristol-Myers Squibb	P1/2	CC (47)
Briquilimab Sickle Cell Disease	National Heart, Lung, and Blood Institute (NHLBI)	P1/2	CC (47)
cd45ra depleted leukocytes Sickle Cell Disease	Paul Szabolcs	P1/2	CC (47)
Biotinylated Human Red Blood Cell Sickle Cell Disease	National Heart, Lung, and Blood Institute (NHLBI)	P1	CC (45)
Autologous Cd34+ Cells Encoding .Beta.A-T87Q-Globin Gene Sickle Cell Disease	Editas Medicine, Inc.	P1/2	CC (44)
Inclacumab Sickle Cell Disease	Pfizer	P3	CC (44)
Midazolam Sickle Cell Disease	Pfizer	P1	CC (44)
Apamistamab Sickle Cell Disease	Columbia University	P1	CC (43)
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)

Showing 51 to 100 of 133 assets

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