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Displaying drugs 12976 - 13000 of 14355 in total
BAL200
Investigational
Puumala virus DNA vaccine
Investigational
Recombinant human CD4 immunoglobulin G
Investigational
Humanized monoclonal antibody Hu11E6
Investigational
Recombinant botulinum A/B vaccine
Investigational
OAV201
OAV201 is a non-replicating, recombinant adeno-associated virus serotype 9 (AAV9) containing the human Methyl CpG Binding Protein 2B (MECP2B) cDNA under the control of a segment of the murine MECP2 promoter. It was first developed by Novartis and investigated for the treatment of Rett syndrome, but further investigation in clinical...
Investigational
Matched Description: … It was first developed by Novartis and investigated for the treatment of Rett syndrome, but further investigation …
AMT-080
AMT-080 is an investigational gene therapy being investigated for the treatment of Duchenne muscular dystrophy.
Investigational
tgAAVCF
tgAAVCF is an adeno-associated virus (AAV) vector encoding the human cystic fibrosis transmembrane conductance regulator protein (CFTR) cDNA. It is being investigated for the treatment of cystic fibrosis.[A259821, A259826]
Investigational
TAK-754
TAK-754 is an adeno-associated virus serotype 8 vector (AAV8) expressing B domain deleted codon optimized factor VIII. Developed by Takeda, it is being investigated for the treatment of hemophilia A.
Investigational
Baliforsen
Investigational
CTx-PDE6b
CTx-PDE6b is an adeno-associated virus (AAV) based gene therapy designed to deliver a full-length non-mutated copy of the functional human PDE6b gene. Being commercialied by Coave Therapeutics, it is being investigated for the treatment of retinitis pigmentosa due to PDE6b gene mutations.
Investigational
MP4CO
Investigational
Skin-derived ABCB5-positive Mesenchymal Stem cells
Skin-derived ABCB5-positive Mesenchymal Stem cells are under investigation in clinical trial NCT04971161 (Allogeneic Abcb5-positive Dermal Mesenchymal Stromal Cells for Treatment of CVU (Phase Iib)).
Investigational
Bacteroides thetaiotaomicron
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.
Investigational
GT-AS
GT-AS recombinant adeno-associated virus serotype 9 vector containing the transgene UBE3A encoding for ubiquitin protein ligase E3A/E6-AP.
Investigational
MANF
MANF is a human recombinant mesencephalic, astrocyte-derived neurotrophic factor.
Investigational
Fazirsiran
Investigational
Prademagene zamikeracel
Investigational
Exon 45 specific phosphorothioate oligonucleotide
Investigational
Exon 52 specific phosphorothioate oligonucleotide
Investigational
SAR422459
SAR422459 is a Lentiviral Vector Containing The Human Abca4 Gene that is currently being investigated to treat Stargardt disease.
Investigational
SAR421869
SAR421869 is a Lentiviral Vector Containing The Human My07A Gene currently being investigated for the treatment of retinitis pigmentosa associated with Usher syndrome 1B gene defect.
Investigational
Sorghum
Investigational
NeuroCell-HD
Investigational
Displaying drugs 12976 - 13000 of 14355 in total