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Displaying drugs 7551 - 7575 of 11962 in total
TOL-101
Investigational
GTX-102
GTX-102 is an antisense oligonucleotide designed to target and inhibit the expression of UBE3A-AS. It is being investigated for the treatment of Angelman Syndrome.
Investigational
MYR-101
MYR-101 (rAAV-Olig001-ASPA) is a recombinant adeno-associated virus (rAAV) vector-based gene therapy intended to treat Canavan disease. Canavan disease is a fatal childhood genetic disorder characterized by white matter degeneration in the brain. It is caused by a mutation in the aspartoacylase gene (ASPA), which leads to a deficiency of the...
Investigational
SBT-100
SBT-100 is an anti-signal transducer and activator of transcription 3 (STAT3) B VHH13 single-domain antibody (sdAb). It was granted orphan drug designation by the FDA for the treatment of pancreatic cancer and osteosarcoma.[L44131,L44136]
Investigational
ICG-140
Investigational
OTL-103
Investigational
RGX-111
RGX-111 is an adeno-associated virus vector AAV9 designed to deliver the α-L-iduronidase (IDUA) gene. Developed by REGENXBIO Inc., it was investigated for the treatment of mucopolysaccharidosis Type I (MPS I).
Investigational
RGX-121
RGX-121 is designed to use the NAV AAV9 vector to deliver the human iduronate-2-sulfatase gene to the central nervous system.
Investigational
AGT-184
Investigational
DTX-101
DTX-101 is an investigational gene therapy consisting of an adeno-associated virus serotype Rh10 vector encoding the human Factor Ix gene. It was previously investigated for the treatment of hemophilia B; however, the clinical development of DTX-101 was discontinued.
Investigational
WP-1301
WP1301 is a synthetic peptide derived from hFVIII. It binds to an MHC class II molecule and is recognized by a Factor VIII-specific T cell.
Investigational
DS-1211
DS-1211 is a potent and selective novel small molecule tissue-nonspecific alkaline phosphatase (TNAP) inhibitor.
Investigational
Matched Iupac: … (2R)-2-acetamido-4-methylpentanoic acid …
AMT-162
AMT-162 is an adeno-associated virus serotype rh10 encoding artificial anti-SOD1 miRNA.
Investigational
DF-1001
Investigational
PTI-110
Investigational
NGN-101
NGN-101 is an adeno-associated virus serotype 9 vector with engineered transgene encoding the human CLN5 gene
Investigational
FBX-101
FBX-101 is an adeno-associated virus serotype rhesus 10 vector expressing the human galactocerebrosidase (GALC) gene
Investigational
TG-1042
TG-1042 (formerly known as ASN-002) is a recombinant adenovirus carrying the cDNA coding sequence for the human interferon gamma gene.
Investigational
CTR-107
CTR-107 is a synthetic targeted growth factor. It is being investigated for the treatment of familial exudative vitreoretinopathy.
Investigational
LMB-100
LMB-100 is a is an antibody-toxin conjugate with an antimesothelin Fab linked to a 24-kilodalton portion of Pseudomonas exotoxin A with mutations that decrease immunogenicity.
Investigational
2-{4-[4-(4-Chloro-Phenoxy)-Benzenesulfonyl]-Tetrahydro-Pyran-4-Yl}-N-Hydroxy-Acetamide
Experimental
Matched Name: … 2-{4-[4-(4-Chloro-Phenoxy)-Benzenesulfonyl]-Tetrahydro-Pyran-4-Yl}-N-Hydroxy-Acetamide …
Matched Iupac: … 2-{4-[4-(4-chlorophenoxy)benzenesulfonyl]oxan-4-yl}-N-hydroxyacetamide …
Matched Iupac: … 2-{4-[4-(4-chlorophenoxy)benzenesulfonyl]oxan-4-yl}-N-hydroxyacetamide …
N-{2'-[(4-FLUOROPHENYL)AMINO]-4,4'-BIPYRIDIN-2-YL}-4-METHOXYCYCLOHEXANECARBOXAMIDE
Experimental
Matched Name: … N-{2'-[(4-FLUOROPHENYL)AMINO]-4,4'-BIPYRIDIN-2-YL}-4-METHOXYCYCLOHEXANECARBOXAMIDE …
Matched Iupac: … (1r,4r)-N-{2'-[(4-fluorophenyl)amino]-[4,4'-bipyridin]-2-yl}-4-methoxycyclohexane-1-carboxamide …
Matched Iupac: … (1r,4r)-N-{2'-[(4-fluorophenyl)amino]-[4,4'-bipyridin]-2-yl}-4-methoxycyclohexane-1-carboxamide …
Displaying drugs 7551 - 7575 of 11962 in total