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Therapeutic Asset Ratings

131 results matching 1 filter

133,990 assets · 8,802 sponsors · 3,128 indications
Methodology v0.2
AssetSponsorPhaseRating
Nusinersen
Spinal Muscular Atrophy
Scholar Rock, Inc.P4A (82)
Viltolarsen
Duchenne Muscular Dystrophy
NS Pharma, Inc.P4BBB (77)
Fitusiran
Hemophilia A
SanofiP4BBB (76)
Tofersen
Amyotrophic Lateral Sclerosis
BiogenP4BBB (72)
Golodirsen
Duchenne Muscular Dystrophy
Rare Disease Research, LLCP4BB (70)
Aoc 1044
DMD
Avidity Biosciences, Inc.P3BB (70)
Plozasiran
Familial Chylomicronemia
Arrowhead PharmaceuticalsP3BB (69)
Avi-4658
Duchenne Muscular Dystrophy
Sarepta Therapeutics, Inc.P3BB (68)
Casimersen
Duchenne Muscular Dystrophy
Kevin FlaniganP3BB (67)
Inclisiran
LDL-Cholersterol Lowering
University of California, San DiegoP4BB (65)
Inclisiran
Primary Prevention
University of California, San DiegoP4BB (65)
Sepofarsen
Retinal Disease
Laboratoires TheaP3BB (65)
Aln-Pnp
Metabolic Dysfunction-Associated Steatotic Liver Disease (MASLD)
Regeneron PharmaceuticalsP2BB (65)
Fitusiran
Hemophilia
SanofiP3BB (64)
all-P-ambo-3'-deoxy-P-dimethylamino-2',3'-imino-2',3'-secothymidylyl-(2’a?5’)-P,3'-dideoxy-P-dimethylamino-2',3'-imino-2',3'-secothymidylyl-(2’a?5’)-P,2',3'-trideoxy-P-dimethylamino-2',3'-imino-2',3'-secoguanylyl-(2’a?5’)-P,3'-dideoxy-P-dimethylamino-2',3'-imino-2',3'-secothymidylyl-(2'a?5')P,2',3'-trideoxy-P-dimethylamino-2',3'-imino-2',3'-secoguanylyl-(2'a?5')-P,3'-dideoxy-P-dimethylamino-2',3'-imino-2',3'-secothymidylyl-(2’a?5’)-P,2',3'-trideoxy-P-dimethylamino-2',3'-imino-2',3'-secocytidylyl-(2’a?5’)-P,3'-dideoxy-P-dimethylamino-2',3'-imino-2',3'-secothymidylyl-(2'a?5')-P,3'-dideoxy-P-dimethylamino-2',3'-imino-2',3'-secothymidylyl-(2’a?5’)-P,3'-dideoxy-P-dimethylamino-2',3'-imino-2',3'-secothymidylyl-(2'a?5')-P,2',3'-trideoxy-P-dimethylamino-2',3'-imino-2',3'-secocytidylyl-(2'a?5')-P,3'-dideoxy-P-dimethylamino-2',3'-imino-2',3'-secothymidylyl-(2'a?5')-P,3'-dideoxy-P-dimethylamino-2',3'-imino-2',3'-secothymidylyl-(2'a?5')-P,2',3'-trideoxy-P-dimethylamino-2',3'-imino-2',3'-secocytidylyl-(2'a?5')-P,3'-dideoxy-P-dimethylamino-2',3'-imino-2',3'-secothymidylyl-(2'a?5')-P,2',3'-trideoxy-P-dimethylamino-2',3'-imino-2',3'-secoguanylyl-(2'a?5')-P,3'-dideoxy-P-dimethylamino-2',3'-imino-2',3'-secothymidylyl-(2'a?5')-P,3'-dideoxy-P-dimethylamino-2',3'-imino-2',3'-secothymidylyl-(2'a?5')-P,2',3'-trideoxy-P-dimethylamino-2',3'-imino-2',3'-secoadenylyl-(2'a?5')-P,2',3'-trideoxy-P-dimethylamino-2',3'-imino-2',3'-secoguanylyl-(2'a?5')-P,2',3'-trideoxy-P-dimethylamino-2',3'-imino-2',3'-secocytidylyl-(2'a?5')-P,2',3'-trideoxy-P-dimethylamino-2',3'-imino-2',3'-secocytidylyl-(2'a?5')-P,2',3'-trideoxy-P-dimethylamino-2',3'-imino-2',3'-secoadenylyl-(2'a?5')-2',3'-trideoxy-2',3'-imino-2',3'-secocytidine
Duchenne Muscular Dystrophy
NS Pharma, Inc.P2B (61)
Aoc 1044
Congenital
Avidity Biosciences, Inc.P3B (60)
Aoc 1044
Genetic Diseases
Avidity Biosciences, Inc.P3B (60)
Aoc 1044
Muscular Disease
Avidity Biosciences, Inc.P3B (60)
Aoc 1044
Muscular Disorders, Atrophic
Avidity Biosciences, Inc.P3B (60)
Aoc 1044
Muscular Dystrophies
Avidity Biosciences, Inc.P3B (60)
Aoc 1044
Musculoskeletal Diseases
Avidity Biosciences, Inc.P3B (60)
Aoc 1044
Neonatal Disease
Avidity Biosciences, Inc.P3B (60)
Aoc 1044
Nervous System Diseases
Avidity Biosciences, Inc.P3B (60)
Aoc 1044
Neuromuscular Diseases (NMD)
Avidity Biosciences, Inc.P3B (60)
Aoc 1044
X-Linked
Avidity Biosciences, Inc.P3B (60)
Showing 1 to 25 of 131 assets
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