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BAX 335
go.drugbank.com/drugs/DB17845InvestigationalBAX 335 is an adeno-associated virus vector 8 (AAV8)–based FIX gene therapy being investigated for the treatment of hemophilia B.[A259791]
Synonyms: AAV8.sc-TTR-FIXR338Lopt, AAV8.ss-3xCRM8-TTRFIX_R338LoptAMT-080
go.drugbank.com/drugs/DB17846ExperimentalAMT-080 is an investigational gene therapy being investigated for the treatment of Duchenne muscular dystrophy.
Flotufolastat F-18
go.drugbank.com/drugs/DB17851ApprovedInvestigationalFlotufolastat F-18 is a diastereoisomer of <sup>18</sup>F-rhPSMA-7, and compared to the other diastereoisomers of this compound, flotufolastat F-18 has a faster clearance from blood pool, liver, and kidney … [Flotufolastat] F-18 (<sup>18</sup>F-rhPSMA-7.3) is an <sup>18</sup>F-labeled ligand that targets the prostate-specific membrane antigen (PSMA).
Synonyms: F-18-rhPSMA-7.3, Flotufolastat F 18AAV-AIPL
go.drugbank.com/drugs/DB17857ExperimentalAAV-AIPL is an adenovirus-associated viral vector serotype 8 containing the human _AIPL1_ gene. … Developed by MeiraGTx, it is being investigated for the treatment of inherited retinal dystrophy due to defects in _AIPL1_ gene.
Synonyms: adenovirus associated viral vector serotype 8 containing the human AIPL1 geneSPL23-2
go.drugbank.com/drugs/DB17866ExperimentalSPL23-2 is an antisense oligonucleotide (ASO) targeting the W1282X nonsense mutation associated with a severe form of cystic fibrosis.[L46767]
WVE-003
go.drugbank.com/drugs/DB17870InvestigationalWVE-003 is an allele-selective antisense oligonucleotide that works to selectively reduce the toxic mutant huntingtin (mHTT) protein.[L46787]
Bacteroides thetaiotaomicron
go.drugbank.com/drugs/DB17879InvestigationalBacteroides thetaiotaomicron is a live biotherapeutic. … Developed by 4D Pharma, Thetanix, an oral formulation comprising lyophilized Bacteroides thetaiotaomicron, is being investigated for the treatment of Crohn’s disease.[A259892]
NGGT-002
go.drugbank.com/drugs/DB17881InvestigationalNGGT-002 is a recombinant adeno-associated virus serotype 8 vector encoding human phenylalanine hydroxylase (PAH).
GT-AS
go.drugbank.com/drugs/DB17883ExperimentalGT-AS recombinant adeno-associated virus serotype 9 vector containing the transgene UBE3A encoding for ubiquitin protein ligase E3A/E6-AP.
Synonyms: GT-ASTAK-603
go.drugbank.com/drugs/DB17892ExperimentalSynonyms: Ethyl 4-(3,4-dimethoxyphenyl)-6,7-dimethoxy-2-(1,2,4-triazol-1-ylmethyl)quinoline-3-carboxylate, 3-quinolinecarboxylic acid, 4-(3,4-dimethoxyphenyl)-6,7-dimethoxy-2-(1h-1,2,4-triazol-1-ylmethyl)-, ethyl esterCategories: Heterocyclic Compounds, 1-Ring, Heterocyclic Compounds, 2-Ring