Search

Displaying results 7751 - 7760 of 10000 in total
  1. Drug

    Ultevursen

    go.drugbank.com/drugs/DB17835
    Investigational

    Ultevursen is a single-stranded RNA-based oligonucleotide targeting exon 13 of the USH2A gene that encodes usherin. … It was developed by ProQR Therapeutics and is being investigated for the treatment of retinitis pigmentosa.

  2. Drug

    AGA2115

    go.drugbank.com/drugs/DB17840
    Investigational

    AGA2115 is a humanized bispecific antibody neutralizing both sclerostin and Dickkopf-1. … Developed by Angitia Biopharmaceuticals, AGA2115 is being investigated for the treatment of osteogenesis imperfect.[L46646]

  3. Drug

    CDX-6512

    go.drugbank.com/drugs/DB17841
    Experimental

    CDX-6512 is a modified, gastrointestinal-stable bacterial leucine decarboxylase. Developed by Codexis, it is being investigated for the treatment of homocystinuria And Maple Syrup Urine Disease.

  4. Drug

    Sonpiretigene isteparvovec

    go.drugbank.com/drugs/DB17844
    Investigational

    Sonpiretigene isteparvovec is an ambient-light activatable Multi-Characteristic Opsin (MCO) optogenetic therapy. … Developed by Nanoscope Therapeutics, it is being investigated for vision restoration in advanced retinitis pigmentosa (RP).

    Synonyms: VMCO-1, vMCO-I
  5. Drug

    BAX 335

    go.drugbank.com/drugs/DB17845
    Investigational

    BAX 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_R338Lopt
  6. Drug

    AMT-080

    go.drugbank.com/drugs/DB17846
    Experimental

    AMT-080 is an investigational gene therapy being investigated for the treatment of Duchenne muscular dystrophy.

  7. Drug

    AAV-AIPL

    go.drugbank.com/drugs/DB17857
    Experimental

    AAV-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 gene
  8. Drug

    SPL23-2

    go.drugbank.com/drugs/DB17866
    Experimental

    SPL23-2 is an antisense oligonucleotide (ASO) targeting the W1282X nonsense mutation associated with a severe form of cystic fibrosis.[L46767]

  9. Drug

    WVE-003

    go.drugbank.com/drugs/DB17870
    Investigational

    WVE-003 is an allele-selective antisense oligonucleotide that works to selectively reduce the toxic mutant huntingtin (mHTT) protein.[L46787]

  10. Drug

    Bacteroides thetaiotaomicron

    go.drugbank.com/drugs/DB17879
    Investigational

    Bacteroides 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]