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Elivaldogene autotemcel
go.drugbank.com/drugs/DB16746ApprovedElivaldogene autotemcel is a gene therapy consisting of genetically modified autologous cells. … [A243691] ALDP is a key protein that normally breaks down fatty substances in the body called very long-chain fatty acids (VLCFAs).
International brands: Lenti-DPlant-based virus-like particles, recombinant, adjuvanted COVID-19 vaccine
go.drugbank.com/drugs/DB16757Approved[L40694] It is composed of VLPs expressing SARS-CoV-2 spike protein trimers (stabilized in a pre-fusion conformation) and is mixed with the AS03 adjuvant system prior to administration. … [A245819] Plant-based VLPs offer a number of advantages as compared to other vaccine platforms, including speed and ease of manufacturing and increased immunogenicity.
Synonyms: Recombinant SARS-CoV-2 spike (S)-protein virus-like particleTyrphostin B44
go.drugbank.com/drugs/DB16762ExperimentalSynonyms: Tyrphostin B-44Categories: Heterocyclic Compounds, 2-RingMorin
go.drugbank.com/drugs/DB16770ExperimentalSynonyms: 2-(2,4-Dihydroxyphenyl)-3,5,7-trihydroxy-4h-1-benzopyran-4-one, Natural yellow 8Categories: Heterocyclic Compounds, 1-Ring, Heterocyclic Compounds, 2-RingRhamnetin
go.drugbank.com/drugs/DB16772ExperimentalSynonyms: 7-O-Methylquercetin, 7-MethoxyquercetinCategories: Heterocyclic Compounds, 1-Ring, Heterocyclic Compounds, 2-RingResamirigene bilparvovec
go.drugbank.com/drugs/DB16779InvestigationalAT132 is under investigation in clinical trial NCT03199469 (Gene Transfer Clinical Study in X-linked Myotubular Myopathy).
Synonyms: DNA (synthetic adeno-associated virus 8 vector AT132 human myotubularin cDNA-specifying), Non-replicating adeno-associated viral vector, serotype 8, expressing human myotubularin geneTefidsogene civaparvovec
go.drugbank.com/drugs/DB16789InvestigationalIt 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
Synonyms: EXONS 2-9 FROM HIDS AND A SPLICE ACCEPTOR SITE (SA), A RECOMBINANT NON-REPLICATING ADENO-ASSOCIATED VIRUS TYPE 2/6 (RAAV REP2-CAP6) VECTOR, WHICH CONTAINS A PROMOTER-LESS HUMAN IDURONATE 2-SULFATASE (HIDS) TRANSGENE CASSETTE, ENCODING PARTS OF EXON 1 PLUSDevafidugene civaparvovec
go.drugbank.com/drugs/DB16790InvestigationalIt uses proprietary zinc finger nucleases (ZFN) that insert a functional copy of the alpha-L-iduronidase (IDUA) enzyme. … Developed by Sangamo Therapeutics, Inc., devafidugene civaparvovec is being investigated for the treatment of mucopolysaccharidosis Type I (MPS I).[L46716]
Delandistrogene moxeparvovec
go.drugbank.com/drugs/DB16802ApprovedInvestigational[L47026] DMD is an X-linked genetic disorder characterized by mutations in the dystrophin gene, leading to a deficiency in functional dystrophin protein. … Microdystrophin is a truncated, functional form of dystrophin.