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ETX101
go.drugbank.com/drugs/DB18579InvestigationalETX101 is a non-replicating recombinant adeno associated viral vector, serotype 9, designed to promote increased transcription of SCN1A gene.
PBFT02
go.drugbank.com/drugs/DB18580InvestigationalPBFT02 is a non-replicating recombinant adeno-associated virus serotype 1 vector delivering human GRN gene encoding the protein progranulin.
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
CSIC002
go.drugbank.com/drugs/DB18584ExperimentalCSIC002 is a small-molecule Sema-3A inhibitor. It is being investigated for the treatment of anterior ischemic optic neuropathy.
Timolumab
go.drugbank.com/drugs/DB18597InvestigationalTimolumab is a 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.
VCA-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'
ATIR101
go.drugbank.com/drugs/DB18650InvestigationalATIR101 is a haploidentical, naïve cell-enriched T-cell therapy.
TUM012
go.drugbank.com/drugs/DB18652InvestigationalTUM012 is a synthetic polymer PEG conjugated to a phospholipid anchor (2-dipalmitoyl-sn-glycerol-3-phosphatidylethanolamine)