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Displaying drugs 14626 - 14650 of 15120 in total
SIG-007 is a gene therapy that consists of cells that are genetically modified with a non-viral vector to express human alpha-galactosidase A. Developed by Sigilon Therapeutics, t is being investigated for the treatment of Fabry Disease.
Investigational
ATX-F8-117 is an investigational peptide derived from Factor VIII. Developed by Apitope, it is being investigated for the treatment of hemophilia A.
Investigational
Investigational
MBP-091 is a monoclonal antibody being investigated for the treatment of Marburg virus disease (MVD) caused by infection with Marburg virus (MARV).
Investigational
Investigational
Investigational
Investigational
Investigational
Investigational
Rivabazumab pegol is being investigated as a treatment of Pseudomonas aeruginosa lung infections in patients with cystic fibrosis.
Investigational
MOR-22, a Natural human lymphoblastoid interferon-alpha, is currently being investigated to treat papillomavirus warts in the oral cavity of HIV-positive patients.
Investigational
INM004, a Neutralizing Equine Anti-Stx Hyperimmune Immunoglobulin F(Ab')2 Fragment, is currently being investigated to treat Shiga-toxin producing bacterial infection relating to the prevention of hemolytic uremic syndrome.
Investigational
VE303 is under clinical development by Vedanta Biosciences.
Investigational
Investigational
Investigational
Investigational
Apc001 is an aptamer developed for the treatment of osteogenesis imperfecta.
Investigational
ENTR-601-44 is an exon 44 skipping phosphorodiamidate morpholino oligonucleotide (PMO). Developed by Entrada Therapeutics, it was investigated for the treatment of Duchenne muscular dystrophy.
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). Sonpiretigene isteparvovec consists of a fusion Adeno-associated virus (AAV) vector with three plasmids encoding opsins with spectrally-separated activation peaks.
Investigational
Investigational
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.
Investigational
QR-313 is an antisense oligonucleotide targeting exon 73. It is being investigated for the treatment of dystrophic epidermolysis bullosa.
Investigational
QRX-411 is a first-in-class RNA-based oligonucleotide designed to restore wild-type USH2A mRNA, leading to the production of the functional USH2A protein. It addresses the underlying cause of Usher syndrome due to the c.7595-2144A>G mutation in the USH2A gene.
Investigational
Displaying drugs 14626 - 14650 of 15120 in total