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Vutrisiran
go.drugbank.com/drugs/DB16699ApprovedInvestigational[L34490,L42065] Vutrisiran is commercially available as a conjugate of N-acetylgalactosamine (GalNAc), a residue that enables the delivery of siRNA to hepatocytes.
Nelistotug
go.drugbank.com/drugs/DB19334InvestigationalNCT06062420 (A Platform Study of Novel Immunotherapy Combinations as First-line Treatment in Participants With PD-L1 Positive Recurrent/metastatic Squamous Cell Carcinoma of the Head and Neck- GALAXIES H&N-
Fluocortolone pivalate
go.drugbank.com/drugs/DB15777InvestigationalMixtures: ULTRAPROCT N, ULTRAPROCT NAnacaulase
go.drugbank.com/drugs/DB17449ApprovedInvestigationalcomposed (80-95% w/w) of proteins such as stem bromelain, ananain, jacalin-like lectin, bromelain inhibitors, and phytocystatin inhibitor, as well as saccharides, as both free monosaccharides and the N-linked
OTL-201
go.drugbank.com/drugs/DB17652ExperimentalIt consists of a lentiviral vector to insert a functional copy of the human N-Sulfoglucosamine Sulfohydrolase (SGSH) gene into autologous CD34+ hematopoietic stem cells.[L45973]
Tralesinidase alfa
go.drugbank.com/drugs/DB17657InvestigationalTralesinidase alfa is a chimeric fusion protein comprised of recombinant human NAGLU and a modified human insulin-like growth factor 2. It is being investigated for the treatment of mucopolysaccharidosis type IIIB (MPS IIIB), also known ...
Synonyms: Alpha-N-acetyglucosaminidase-insulin-like-growth-factor-2 fusion protein, Recombinant human alpha-N-acetylglucosaminidase-insulin-like growth factor-2Categories: beta-N-AcetylhexosaminidasesGivosiran
go.drugbank.com/drugs/DB15066ApprovedGivosiran is a small interfering RNA (siRNA) directed towards 5-aminolevulinic acid synthase, a critical enzyme in the heme biosynthesis pathway. It is manufactured by Alnylam Pharmaceuticals and was first approved for use in the United ...
Tovorafenib
go.drugbank.com/drugs/DB15266ApprovedInvestigationalTovorafenib is a selective type II RAF kinase inhibitor with anti-tumour activity. It was granted accelerated approval by the FDA, making it the first FDA approval of a systemic therapy for the treatment of pediatric low-grade glioma, wi...
Synonyms: 4-Pyrimidinecarboxamide, 6-amino-5-chloro-N-[(1R)-1-[5-[[[5-chloro-4-(trifluoromethyl)-2-pyridinyl]amino]carbonyl]-2-thiazolyl]ethyl]-, 6-amino-5-chloro-N-[1R)-1-[5-[[[5-hloro-4-(trifluoromethyl)-2pyridinyl]amino]carbonyl]-2-thiazoyl]ethyl]-4-pyrimdinecarboxamide