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Displaying drugs 2001 - 2025 of 3550 in total
RP-A501
RP-A501 is a gene therapy product consisting of a recombinant adeno-associated virus serotype 9 (rAAV9) capsid containing the human LAMP2B transgene (AAV9:LAMP2B), which encodes lysosome-associated membrane protein 2 (LAMP-2). It is being investigated for Danon disease.
Investigational
Matched Synonyms: … Adeno-associated virus serotype 9 vector containing the human lysosome-associated membrane glycoprotein …
Matched Description: … RP-A501 is a gene therapy product consisting of a recombinant adeno-associated virus serotype 9 (rAAV9 …
Matched Description: … RP-A501 is a gene therapy product consisting of a recombinant adeno-associated virus serotype 9 (rAAV9 …
Dimethylaminomicheliolide
Investigational
Matched Synonyms: … AZULENO(4,5-B)FURAN-2(3H)-ONE, 3-((DIMETHYLAMINO)METHYL)-3A,4,5,7,8,9,9A,9B-OCTAHYDRO-9-HYDROXY-6,9-DIMETHYL …
Matched Iupac: … (3R,3aS,9R,9aS,9bS)-3-[(dimethylamino)methyl]-9-hydroxy-6,9-dimethyl-2H,3H,3aH,4H,5H,7H,8H,9H,9aH,9bH-azuleno …
Matched Iupac: … (3R,3aS,9R,9aS,9bS)-3-[(dimethylamino)methyl]-9-hydroxy-6,9-dimethyl-2H,3H,3aH,4H,5H,7H,8H,9H,9aH,9bH-azuleno …
TH-9402 free base
Investigational
Matched Synonyms: … Benzoic acid, 2-(6-amino-4,5-dibromo-3-imino-3h-xanthen-9-yl)-, methyl ester …
Matched Iupac: … methyl 2-(6-amino-4,5-dibromo-3-imino-3H-xanthen-9-yl)benzoate …
Matched Iupac: … methyl 2-(6-amino-4,5-dibromo-3-imino-3H-xanthen-9-yl)benzoate …
Certepetide
Investigational
Matched Synonyms: … Ac-Cys-Arg-Gly-Asp-Lys-Gly-Pro-Asp-Cys-NH2 cyclic (1→9)-disulfide ... , N-acetyl-L-cysteinyl-L-arginylglycyl-L-α-aspartyl-L-lysylglycyl-L-prolyl-L-α-aspartyl-, cyclic (1→9) ... N-Acetyl-L-cysteinyl-L-arginylglycyl-L-α-aspartyl-L-lysylglycyl-L-prolyl-L-α-aspartyl-L-cysteinamide, cyclic (1→9) …
2'-deoxythioguanosine
Investigational
Matched Synonyms: … 2-amino-9-(2-deoxy-.beta.-d-erythro-pentofuranosyl)-6-mercaptopurine …
Matched Iupac: … 2-amino-9-[(2R,4S,5R)-4-hydroxy-5-(hydroxymethyl)oxolan-2-yl]-6,9-dihydro-1H-purine-6-thione …
Matched Iupac: … 2-amino-9-[(2R,4S,5R)-4-hydroxy-5-(hydroxymethyl)oxolan-2-yl]-6,9-dihydro-1H-purine-6-thione …
QN-302
Investigational
Matched Synonyms: … 2,7-bis(3-morpholinopropyl)-4-((2-(pyrrolidin-1-yl)ethyl)amino)-9-(4-(pyrrolidin-1-ylmethyl)phenyl)benzo …
Matched Iupac: … 6,13-bis[3-(morpholin-4-yl)propyl]-2-{[2-(pyrrolidin-1-yl)ethyl]amino}-9-{4-[(pyrrolidin-1-yl)methyl] …
Matched Iupac: … 6,13-bis[3-(morpholin-4-yl)propyl]-2-{[2-(pyrrolidin-1-yl)ethyl]amino}-9-{4-[(pyrrolidin-1-yl)methyl] …
KTX-1001
Investigational
Matched Synonyms: … (S)-1-((R)-3-amino-1-(4-((6-amino-9H-purin-9-yl)methyl)-6-(2,5-difluoro-4-methoxyphenyl)pyridin-3-yl) …
AAV9/MFSD8
AAV9/MFSD8 is a gene therapy developed by Neurogene Inc. It is an adeno-associated virus serotype 9 (AAV9) vector with an engineered transgene encoding the human CLN7/MFSD8 gene for expression of active human major facilitator superfamily domain containing 8. It was investigated for the treatment of neuronal ceroid lipofuscinosis type 7.
Investigational
Matched Synonyms: … An adeno-associated virus serotype 9 (AAV9) vector with engineered transgene encoding the human CLN7/ …
Matched Description: … It is an adeno-associated virus serotype 9 (AAV9) vector with an engineered transgene encoding the human …
Matched Description: … It is an adeno-associated virus serotype 9 (AAV9) vector with an engineered transgene encoding the human …
Tafolecimab
Tafolecimab is under investigation in clinical trial NCT04031742 (A Study to Evaluate Safety and Efficacy of IBI306, a PCSK9 Monoclonal Antibody in Chinese Subjects With Homozygous Familial Hypercholesterolemia).
Investigational
Matched Synonyms: … Immunoglobulin igg2, anti-(human proprotein convertase subtilisin/kexin type 9) (human monoclonal ibi306 …
Pinometostat
Pinometostat has been used in trials studying the treatment of Leukemia, Acute Leukemias, Acute Myeloid Leukemia, Myelodysplastic Syndrome, and Acute Lymphocytic Leukemia, among others.
Investigational
Matched Synonyms: … 9H-PURIN-6-AMINE, 9-(5-DEOXY-5-((CIS-3-(2-(6-(1,1-DIMETHYLETHYL)-1H-BENZIMIDAZOL-2-YL)ETHYL)CYCLOBUTYL …
Matched Iupac: … (2R,3R,4S,5R)-2-(6-amino-9H-purin-9-yl)-5-{[(propan-2-yl)[(1r,3s)-3-[2-(6-tert-butyl-1H-1,3-benzodiazol …
Matched Iupac: … (2R,3R,4S,5R)-2-(6-amino-9H-purin-9-yl)-5-{[(propan-2-yl)[(1r,3s)-3-[2-(6-tert-butyl-1H-1,3-benzodiazol …
Tagetitoxin
Tagetitoxin is a bacterial phytotoxin. It preferentially inhibits eukaryotic RNA polymerase.
Experimental
Matched Synonyms: … (1R,4R,5R,6R,7S,8R)-6-Acetoxy-7-amino-4-carbamoyl-4-hydroxy-8-(phosphonooxy)-9-oxa-3-thiabicyclo[3.3.1 …
Matched Iupac: … (1R,4R,5R,6R,7S,8R)-6-(acetyloxy)-7-amino-4-carbamoyl-4-hydroxy-8-(phosphonooxy)-9-oxa-3-thiabicyclo[ …
Matched Iupac: … (1R,4R,5R,6R,7S,8R)-6-(acetyloxy)-7-amino-4-carbamoyl-4-hydroxy-8-(phosphonooxy)-9-oxa-3-thiabicyclo[ …
[(2R,3S,4R,5R)-5-(4-acetonyl-3-carbamoyl-pyridin-1-ium-1-yl)-3,4-dihydroxy-tetrahydrofuran-2-yl]methyl [[(2R,3S,4R,5R)-5-(6-aminopurin-9-yl)-3,4-dihydroxy-tetrahydrofuran-2-yl]methoxy-hydroxy-phosphoryl] phosphate
Experimental
Matched Name: … carbamoyl-pyridin-1-ium-1-yl)-3,4-dihydroxy-tetrahydrofuran-2-yl]methyl [[(2R,3S,4R,5R)-5-(6-aminopurin-9- …
Matched Iupac: … 1-[(2R,3R,4S,5R)-5-{[({[(2R,3S,4R,5R)-5-(6-amino-9H-purin-9-yl)-3,4-dihydroxyoxolan-2-yl]methoxy}(hydroxy …
Matched Iupac: … 1-[(2R,3R,4S,5R)-5-{[({[(2R,3S,4R,5R)-5-(6-amino-9H-purin-9-yl)-3,4-dihydroxyoxolan-2-yl]methoxy}(hydroxy …
NGN-401
NGN-401 is a recombinant serotype 9 adeno-associated virus (AAV) encoding an intron encoding a miRNA generating sequence, complementary miRNA binding sites and a full-length human methyl cytosine binding protein 2 (MECP2). Developed by Neurogene Inc., it is being investigated for the treatment of Rett syndrome.
Investigational
Matched Synonyms: … Recombinant serotype 9 adeno-associated virus (AAV) encoding an intron encoding a miRNA generating sequence …
Matched Description: … NGN-401 is a recombinant serotype 9 adeno-associated virus (AAV) encoding an intron encoding a miRNA …
Matched Description: … NGN-401 is a recombinant serotype 9 adeno-associated virus (AAV) encoding an intron encoding a miRNA …
L-377202
L-377202 is under investigation in clinical trial NCT00987753 (Study Evaluating the Safety and Tolerability of L-377202).
Investigational
Matched Synonyms: … 5,12-NAPHTHACENEDIONE, 10-((3-(((4R)-1-(4-CARBOXY-1-OXOBUTYL)-4-HYDROXY-L-PROLYL-L-ALANYL-L-SERYL-(2S ... hydroxymethyl)-2-(((1s)-1-(((2s,3s,4s,6r)-3-hydroxy-2-methyl-6-(((1s,3s)-3,5,12-trihydroxy-3-(2-hydroxyacetyl)-10 …
Minnelide free acid
Investigational
Matched Synonyms: … Trisoxireno(4b,5:6,7:8a,9)phenanthro(1,2-c)furan-1(3h)-one, 3b,4,4a,6,6a,7a,7b,8b,9,10-decahydro-8b-methyl …
5'-S-methyl-5'-thioadenosine
Experimental
Matched Synonyms: … 9-(5-S-methyl-5-thio-β-D-ribofuranosyl)-9H-purin-6-amine …
Matched Iupac: … (2R,3R,4S,5S)-2-(6-amino-9H-purin-9-yl)-5-[(methylsulfanyl)methyl]oxolane-3,4-diol …
Matched Iupac: … (2R,3R,4S,5S)-2-(6-amino-9H-purin-9-yl)-5-[(methylsulfanyl)methyl]oxolane-3,4-diol …
Ceramide NP
Ceramide NP is a lipid molecule included in a group of lipid molecules called ceramides. Ceramides are major lipid components in the stratum corneum of the human skin. Ceramide 3 consists of a phytosphingosine backbone N-acylated with a saturated fatty acid (stearic acid) . It is widely used as a...
Experimental
Matched Synonyms: … Cer(t18:0/18:1n-9) …
Matched Iupac: … (9Z)-N-[(2S,3S,4R)-1,3,4-trihydroxyoctadecan-2-yl]octadec-9-enamide …
Matched Iupac: … (9Z)-N-[(2S,3S,4R)-1,3,4-trihydroxyoctadecan-2-yl]octadec-9-enamide …
S-adenosyl-L-homocysteine
5'-S-(3-Amino-3-carboxypropyl)-5'-thioadenosine. Formed from S-adenosylmethionine after transmethylation reactions.
Experimental
Matched Synonyms: … S-[1-(adenin-9-yl)-1,5-dideoxy-β-D-ribofuranos-5-yl]-L-homocysteine …
Matched Iupac: … (2S)-2-amino-4-({[(2S,3S,4R,5R)-5-(6-amino-9H-purin-9-yl)-3,4-dihydroxyoxolan-2-yl]methyl}sulfanyl)butanoic …
Matched Iupac: … (2S)-2-amino-4-({[(2S,3S,4R,5R)-5-(6-amino-9H-purin-9-yl)-3,4-dihydroxyoxolan-2-yl]methyl}sulfanyl)butanoic …
Fazirsiran
Investigational
Matched Synonyms: … RNA, ((1'-DE(6-AMINO-9H-PURIN-9-YL))DA-(5'->5')-SP-AM-GM-CM-GM-UM-UM- UM-AM-(2'-DEOXY-2'-FLUORO)G-(2' ... '-FLUORO)G-(2'-DEOXY-2'-FLUORO)C-AM- UM-GM-UM-UM-UM-AM-AM-CM-AM-(3'->3')-SP-(1'-DE(6-AMINO-9H-PURIN-9- …
AVA-6000
Investigational
Matched Synonyms: … N-[(2R)-1-[(2S)-2-[(3-hydroxy-2-methyl-6-{[(3S)-3,5,12-trihydroxy-3-(2-hydroxyacetyl)-10-methoxy-6,11 …
Cardiopet
Cardiopet is under investigation in clinical trial NCT01826773 (Cardiopet as PET Imaging Agent to Assess Myocardial Perfusion and Fatty Acid Uptake in Known or Suspected CAD Subjects).
Investigational
Matched Synonyms: … Trans-9(rs)-18f-fluoro-3,4(rs,rs)-methyleneheptadecanoic acid …
Minnelide
Investigational
Matched Synonyms: … Trisoxireno(4b,5:6,7:8a,9)phenanthro(1,2-c)furan-1(3h)-one, 3b,4,4a,6,6a,7a,7b,8b,9,10-decahydro-8b-methyl …
Methyl bacteriopurpurinimide
Investigational
Matched Synonyms: … 9,12-imino-2,21-metheno-7,4:14,17-dinitrilo-4h-pyrido(4,3-b)azacyclononadecine-16-propanoic acid, 10- …
Glyceryl 1-oleate
Experimental
Matched Synonyms: … 9-Octadecenoic acid, 2,3-dihydroxypropyl ester …
Matched Iupac: … 2,3-dihydroxypropyl (9Z)-octadec-9-enoate …
Matched Iupac: … 2,3-dihydroxypropyl (9Z)-octadec-9-enoate …
Displaying drugs 2001 - 2025 of 3550 in total