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Displaying drugs 11551 - 11575 of 12789 in total
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.
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.
Investigational
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.[L46651, L46656]
Investigational
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
AMT-080 is an investigational gene therapy being investigated for the treatment of Duchenne muscular dystrophy.
Investigational
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 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
Investigational
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
Investigational
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
Bifidobacterium Longum Infantis 35624 is a probiotic being investigated for the treatment of gastrointestinal disorders, such as Crohn's disease and irritable bowel syndrome.[A259872, A259877]
Investigational
Dabocemagene autoficel is an ex vivo, autologous cell-based gene therapy. It comprises autologous dermal fibroblasts genetically modified to express functional type VII collagen. Developed by Castle Creek Biosciences Inc., dabocemagene autoficel is being investigated for the treatment of dystrophic epidermolysis bullosa.
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.
Investigational
GT-AS recombinant adeno-associated virus serotype 9 vector containing the transgene UBE3A encoding for ubiquitin protein ligase E3A/E6-AP.
Investigational
MANF is a human recombinant mesencephalic, astrocyte-derived neurotrophic factor.
Investigational
jCell is an human retinal progenitor cell therapy for retinitis pigmentosa.
Investigational
SAR422459 is a Lentiviral Vector Containing The Human Abca4 Gene that is currently being investigated to treat Stargardt disease.
Investigational
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
Investigational
Investigational
Displaying drugs 11551 - 11575 of 12789 in total