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Displaying drugs 1351 - 1375 of 2785 in total
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 …
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 …
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] …
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) …
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 …
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[ …
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 …
The insulin-like growth factor II is a well-characterized neutral peptide believed to be secreted by the liver and to circulate in the blood. This factor presents effects related to growth regulation, insulin-like and mitogenic activities. To perform its biological activities, this growth factor can present some dependence to somatotropin. It...
Experimental
Matched Synonyms: … Human oligopeptide-24
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 …
Investigational
Matched Synonyms: … -aspartyl-, cyclic (1->9)-disulfide …
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 …
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 …
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- …
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 …
Experimental
Matched Synonyms: … 9-Octadecenoic acid, 2,3-dihydroxypropyl ester …
Matched Iupac: … 2,3-dihydroxypropyl (9Z)-octadec-9-enoate …
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 …
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 …
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 …
Withdrawn
Matched Synonyms: … 5-(Dimethylamino)-9-methyl-2-propyl-1H-pyrazolo(1,2-a)(1,2,4)benzotriazine-1,3(2H)-dione …
Tefidsogene civaparvovec, formerly known as SB-913, is an investigational therapy for genome editing. It consists of an adeno-associated virus (AAV) type 2/6 vector and a zinc finger nuclease (ZFN) system that inserts a correct copy of the Iduronate 2-Sulfatase (IDS) gene into the albumin locus in hepatocytes. Developed by Sangamo...
Experimental
Matched Synonyms: … PROMOTER-LESS HUMAN IDURONATE 2-SULFATASE (HIDS) TRANSGENE CASSETTE, ENCODING PARTS OF EXON 1 PLUS EXONS 2-9
An analog of inosine with a methylthio group replacing the hydroxyl group in the 6-position.
Experimental
Matched Synonyms: … 6-methyl-9-ribofuranosylpurine-6-thiol …
Matched Iupac: … (2R,3S,4R,5R)-2-(hydroxymethyl)-5-[6-(methylsulfanyl)-9H-purin-9-yl]oxolane-3,4-diol …
Ociperlimab is under investigation in clinical trial NCT04866017 (A Study to Compare Ociperlimab Plus Tislelizumab Versus Durvalumab Following Concurrent Chemoradiotherapy (Ccrt) in Patients With Stage III Unresectable Non-small Cell Lung Cancer).
Investigational
Matched Synonyms: … g1-kappa, anti-(homo sapiens tigit (t-cell immunoreceptor with ig domain and itim, v-set ig member 9, …
Bemnifosbuvir is under investigation in clinical trial NCT05629962 (SUNRISE-3: Efficacy and Safety of Bemnifosbuvir in High-risk Outpatients With COVID-19).
Investigational
Matched Synonyms: … (s)-isopropyl 2-((s)-(((2r,3r,4r,5r)-5-(2-amino-6-(methylamino)-9h-purin-9-yl)-4-fluoro-3-hydroxy-4-methyltetrahydrofuran …
Investigational
Matched Salts cas: … 2255306-73-9
Displaying drugs 1351 - 1375 of 2785 in total