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Displaying drugs 776 - 800 of 1753 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 …
Pristimerin
Pristimerin is a quinone methide triterpenoid researched for its anti-cancer potential.
Investigational
Matched Synonyms: … (9.beta.,13.alpha.,14.beta.,20.alpha.)-3-hydroxy-9,13-dimethyl-2-oxo-24,25,26-trinoroleana-1(10),3,5,7 ... 24,25,26-trinoroleana-1(10),3,5,7-tetraen-29-oic acid, 3-hydroxy-9,13-dimethyl-2-oxo-, methyl ester, (9. …
7.beta.-hydroxycholesteryl-3-oleate
Investigational
Matched Synonyms: … Cholest-5-ene-3,7-diol, 3-(9z)-9-octadecenoate, (3.beta.,7.beta.)- …
Matched Iupac: … yl]-1H,2H,3H,3aH,3bH,4H,6H,7H,8H,9H,9aH,9bH,10H,11H,11aH-cyclopenta[a]phenanthren-7-yl (9Z)-octadec-9- …
Matched Iupac: … yl]-1H,2H,3H,3aH,3bH,4H,6H,7H,8H,9H,9aH,9bH,10H,11H,11aH-cyclopenta[a]phenanthren-7-yl (9Z)-octadec-9- …
Ongericimab
Ongericimab is under investigation in clinical trial NCT05325203 (A Study to Evaluate the Efficacy and Safety of JS002 in Patients With Heterozygous Familial Hypercholesterolemia (Hefh).).
Investigational
Matched Synonyms: … Immunoglobulin g4-kappa, anti-(homo sapienspcsk9 (proprotein convertase subtilisin/kexin type 9, neural ... apoptosis-regulated convertase 1, narc1, narc-1, proprotein convertase 9, pc9)), humanized monoclonal …
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 …
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 …
EDIT-101
EDIT-101 is a recombinant adeno-associated virus pseudotyped with serotype 5 viral capsid encoding CRISPR/Cas9 (clustered regularly interspaced short palindromic repeats associated protein 9) gene editing constructs.
Investigational
Matched Synonyms: … encoding CRISPR/Cas9 (clustered regularly interspaced short palindromic repeats associated protein 9) …
Matched Description: … encoding CRISPR/Cas9 (clustered regularly interspaced short palindromic repeats associated protein 9) …
Matched Description: … encoding CRISPR/Cas9 (clustered regularly interspaced short palindromic repeats associated protein 9) …
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] …
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 …
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) …
VPO-227
Investigational
Matched Synonyms: … (+-)-9-(5-bromofuran-2-yl)-12,14-dimethyl-13,15-dioxo-17-phenyl-8-oxa-1,12,14-triazatetracyclo[8.7.0.02,7.011,16 …
Matched Iupac: … 9-(5-bromofuran-2-yl)-12,14-dimethyl-13,15-dioxo-17-phenyl-8-oxa-1,12,14-triazatetracyclo[8.7.0.0^{2,7 …
Matched Iupac: … 9-(5-bromofuran-2-yl)-12,14-dimethyl-13,15-dioxo-17-phenyl-8-oxa-1,12,14-triazatetracyclo[8.7.0.0^{2,7 …
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 …
Lenabasum
Ajulemic acid has been used in trials studying the treatment of Cystic Fibrosis, Dermatomyositis, and Diffuse Cutaneous Systemic Sclerosis.
Investigational
Matched Synonyms: … (6aR,10aR)-1-hydroxy-6,6-dimethyl-3-(2-methyloctan-2-yl)-6a,7,10,10a-tetrahydrobenzo[c]chromene-9-carboxylic …
Matched Iupac: … (6aR,10aR)-1-hydroxy-6,6-dimethyl-3-(2-methyloctan-2-yl)-6H,6aH,7H,10H,10aH-benzo[c]isochromene-9-carboxylic …
Matched Iupac: … (6aR,10aR)-1-hydroxy-6,6-dimethyl-3-(2-methyloctan-2-yl)-6H,6aH,7H,10H,10aH-benzo[c]isochromene-9-carboxylic …
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 …
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[ …
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 …
Metacavir
Metacavir is under investigation in clinical trial NCT02965859 (The Safety and Dose-range Study of Metacavir Enteric-coated Capsules in Patients With Chronic Hepatitis B).
Investigational
Matched Synonyms: … ((2s,5r)-5-(2-amino-6-methoxy-purin-9-yl)tetrahydrofuran-2-yl)methanol …
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 …
[(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 …
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 …
Lerodalcibep
Lerodalcibep is under investigation in clinical trial NCT05004675 (Trial to Evaluate Efficacy and Safety of LIB003 and Inclisiran in High-risk CVD Patients).
Investigational
Matched Synonyms: … Engineered binding protein (1-96) anti-(human proprotein convertase subtilisin/kexin type 9), derived ... Protein lib003 (recombinant human proprotein convertase subtilisin/kexin type 9 ligand-binding tenth …
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 …
Celastrol
Celastrol is a pentacyclic triterpenoid isolated from the root extracts of Tripterygium wilfordii.
Investigational
Matched Synonyms: … 24,25,26-trinoroleana-1(10),3,5,7-tetraen-29-oic acid, 3-hydroxy-9,13-dimethyl-2-oxo-, (9.beta.,13.alpha …
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- …
Displaying drugs 776 - 800 of 1753 in total