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Displaying drugs 1926 - 1950 of 3208 in total
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 …
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 …
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- …
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 …
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] …
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 …
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) …
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 …
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 …
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 …
Investigational
Matched Synonyms: … -aspartyl-, cyclic (1->9)-disulfide …
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[ …
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 …
Investigational
Matched Synonyms: … 20-mer complementary to Akt mRNA …
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 …
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 …
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 …
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 …
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- …
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 …
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 …
Experimental
Matched Synonyms: … 9-Octadecenoic acid, 2,3-dihydroxypropyl ester …
Matched Iupac: … 2,3-dihydroxypropyl (9Z)-octadec-9-enoate …
Investigational
Matched Synonyms: … monoclonal SHR-1316 .gamma.4-chain) fusion protein with peptide linker ((ggggs)4g) fusion protein with 20
Investigational
Matched Synonyms: … NLX-101
Investigational
Matched Synonyms: … gamma2 heavy chain Homo sapiens (1-444) [VH(Homo sapiens IGHV3-20*01 (98.0%) -(IGHD)-IGHJ6*01 (93.8%) …
Displaying drugs 1926 - 1950 of 3208 in total