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scAAV9.U7.ACCA
go.drugbank.com/drugs/DB18582InvestigationalscAAV9.U7.ACCA is a non-replicating recombinant adeno-associated virus vector containing 4 antisense sequences designed to target exon 2 in the human DMD gene under the control of U7 small nuclear non-coding
Synonyms: non-replicating recombinant adeno-associated virus vector containing 4 antisense sequences designed to target exon 2 in the human DMD gene under the control of U7 small nuclear non-coding RNACSIC002
go.drugbank.com/drugs/DB18584ExperimentalCSIC002 is a small-molecule Sema-3A inhibitor. It is being investigated for the treatment of anterior ischemic optic neuropathy.
NGN-401
go.drugbank.com/drugs/DB18594Investigationalprotein 2 (MECP2). … 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
Synonyms: 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 2Timolumab
go.drugbank.com/drugs/DB18597InvestigationalTimolumab is a recombinant, fully-human, IgG4 immunoglobulin with antigen-binding specificity for human vascular adhesion protein-1 (VAP-1).
Synonyms: recombinant, fully-human, IgG4 immunoglobulin with antigen-binding specificity for human vascular adhesion protein-1 (VAP-1)TSHA-102
go.drugbank.com/drugs/DB18599InvestigationalTSHA-102 is a recombinant serotype 9 adeno-associated virus encoding a codon-optimized human MECP2 gene.
TPX-4589
go.drugbank.com/drugs/DB18604InvestigationalTPX-4589 is an antibody-drug conjugate composed of a humanized monoclonal antibody targeting CLDN18.2 and linker-payload MC-VC-PAB-MMAE.
Synonyms: Antibody drug conjugate composed of a humanized monoclonal antibody targeting CLDN18.2 and linker-payload MC-VC-PAB-MMAEVCA-894A
go.drugbank.com/drugs/DB18609InvestigationalVCA-894A is a synthetic 2'-O-methoxyethyl phosphorothioate oligoribonucleotide sodium salt consisting of 19 nucleotide residues with the sequence 5'-MeCMeUGMeUGGAAGMeUGAGGGMeCMeCAG-3'
PRG-A01
go.drugbank.com/drugs/DB18613InvestigationalPRG-A01 is a novel inhibitor of Cu, Zn-superoxide dismutase (SOD1) misfolding and aggregation.
ABBV-011
go.drugbank.com/drugs/DB18622InvestigationalABBV-011 is an antibody-drug conjugate of humanized, cysteine-engineered IgG1 monoclonal antibody targeting seizure-related 6 homolog conjugated to N-acetyl-gamma-calicheamicin.
Synonyms: an antibody-drug conjugate of humanized, cysteine-engineered IgG1 monoclonal antibody targeting seizure-related 6 homolog conjugated to N-acetyl-gamma-calicheamicin