Therapeutic Asset Ratings
464 results matching 1 filter
129,767 assets · 8,707 sponsors · 3,128 indications
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Asset
Sponsor
Phase
Rating
Pelareorep
Anal Carcinoma
Oncolytics Biotech
P1/2
CCC
(48)
Pelareorep
Anal Squamous Cell Carcinoma
Oncolytics Biotech
P1/2
CCC
(48)
Pelareorep
Exocrine Pancreatic Carcinoma
Oncolytics Biotech
P1/2
CCC
(48)
Pexastimogene Devacirepvec
Metastatic Melanoma
Lee's Pharmaceutical Limited
P1/2
CCC
(48)
sb-913
Mucopolysaccharidosis Type 2
Sangamo Therapeutics
P1/2
CCC
(48)
Tgx-007
Malignant Glioma
Trogenix ltd
P1/2
CCC
(48)
Tilt-123
Ovarian Carcinoma
TILT Biotherapeutics Ltd.
P1/2
CCC
(48)
Tsha-105
Developmental And Epileptic Encephalopathy, 25
TESS Research Foundation
P1/2
CCC
(48)
Vgn-R08B
Gaucher Disease Type 3
Shanghai Vitalgen BioPharma Co., Ltd.
P1/2
CCC
(48)
Double Recombinant Vaccinia Virus Vv-Gmcsf-Lact
Oncolytic Virotherapy
"Oncostar" LLC
P1
CC
(47)
G 207
Primitive Neuroectodermal Tumor
M.D. Anderson Cancer Center
P1
CC
(47)
ab-1009
Glycogen Storage Disease Due To Acid Maltase Deficiency, Late-Onset
AskBio Inc
P1/2
CC
(47)
Alnugranogene Aldeparvovec
Frontotemporal Dementia
Passage Bio, Inc.
P1/2
CC
(47)
AT845
Glycogen Storage Disease Due To Acid Maltase Deficiency, Late-Onset
Astellas Gene Therapies
P1/2
CC
(47)
Bmn 331
Hereditary Angioedema
BioMarin Pharmaceutical
P1/2
CC
(47)
DNA (recombinant adeno-associated virus vector AAV-Spark100-GAA human liver-specific expression cassette including human lysosomal ?-glucosidase codon-optimized gene GAA plus adeno-associated virus 2 inverted terminal repeat flanks)
Glycogen Storage Disease II
Spark Therapeutics, Inc.
P1/2
CC
(47)
DNA (recombinant adeno-associated virus vector AAV-Spark100-GAA human liver-specific expression cassette including human lysosomal ?-glucosidase codon-optimized gene GAA plus adeno-associated virus 2 inverted terminal repeat flanks)
Glycogen Storage Disease Due To Acid Maltase Deficiency, Late-Onset
Spark Therapeutics, Inc.
P1/2
CC
(47)
DNA (RECOMBINANT HUMAN HEMATOPOIETIC STEM CELL-DERIVED ADENO-ASSOCIATED VIRUS VECTOR SEROTYPE AAVHSC15 HUMAN LIVER-SPECIFIC EXPRESSION CASSETTE INCLUDING HUMAN PHENYLALANINE 4-HYDROXYLASE CODON-OPTIMIZED CDNA PLUS ADENO-ASSOCIATED VIRUS 2 INVERTED TERMINAL REPEAT FLANKS)
Phenylketonuria
Homology Medicines, Inc
P1/2
CC
(47)
DNA (recombinant replication-defective adeno-associated virus serotype 9 vector UX701 murine transthyretin enhancer plus human transthyretin promoter plus simian virus 40 intron plus human adenosine triphosphatase copper-transporting ? metal-binding domain 4/5/6 transgene ATP7B-MBD456 plus simian virus 40 polyadenylation signal plus adeno-associated virus 2 inverted terminal repeat flanks plus adeno-associated virus 2 Rep region plus adeno-associated virus 9 Cap region-specifying)
Wilson Disease
Ultragenyx Pharmaceutical Inc
P1/2
CC
(47)
DNA (RECOMBINANT REPLICATION-DEFICIENT ADENO-ASSOCIATED VIRUS 5 VECTOR BMN 307 HUMAN LIVER-SPECIFIC TRANSCRIPTION REGULATORY ELEMENT PLUS HUMAN PHENYLALANINE 4-HYDROXYLASE CODON-OPTIMIZED GENE HPAH PLUS ADENO-ASSOCIATED VIRUS 2 INVERTED TERMINAL REPEAT FLANKS)
Phenylketonuria
BioMarin Pharmaceutical
P1/2
CC
(47)
Duvalgagene Otiparvovec
Fabry Disease
4D Molecular Therapeutics
P1/2
CC
(47)
etx101
Dravet Syndrome
Encoded Therapeutics
P1/2
CC
(47)
gc101
Spinal Muscular Atrophy
GeneCradle Inc
P1/2
CC
(47)
gc101
Spinal Muscular Atrophy, Type III
GeneCradle Inc
P1/2
CC
(47)
gc301
Glycogen Storage Disease Due To Acid Maltase Deficiency, Late-Onset
GeneCradle Inc
P1/2
CC
(47)
Showing 176 to 200 of 464 assets
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