Displaying drugs 15301 - 15325 of 15564 in total
VTX-801
VTX-801 is a replication-deficient recombinant adeno-associated viral vector (rAAV) consisting of an AAV liver tropic capsid containing a single-stranded DNA genome carrying a shortened version of the ATP7B gene (ATP7B-minigene). It is being investigated in Wilson Disease.
Investigational
EGT-201
EGT-201 is an adeno-associated viral vector serotype 9 (AAV9) vector-based gene therapy consisting of the alpha-N-acetyglucosaminidase (NAGLU) transgene. Developed by Esteve Pharmaceuticals, it is being investigated for Sanfilippo syndrome type B.
Investigational
ACG-801
Investigational
Pegtibatinase
Investigational
PRX-OTC
PRX-OTC is an intracellular enzyme replacement therapy designed to replace missing or defective enzyme in patients with ornithine transcarbamylase deficiency.
Investigational
ARCT-810
ARCT-810 is an mRNA replacement therapy that delivers ornithine transcarbamylase mRNA to hepatocytes.
Investigational
SAR445136
SAR445136 is an investigational zinc finger nuclease gene-edited cell therapy. It consists of autologous CD34+ hematopoietic stem precursor cells (HSPC) modified ex vivo by zinc finger nucleases (ZFN) that target the BCL11A gene erythroid-specific enhancer (ESE) to increase endogenous HbF production in erythrocytes (RBC). It was formerly investigated for the...
Investigational
Matched Description: … [A259063] It was formerly investigated for the treatment of beta-thalassemia, and it is currently being …
SIG-007
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
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
Nix-0699
Investigational
MBP-091
MBP-091 is a monoclonal antibody being investigated for the treatment of Marburg virus disease (MVD) caused by infection with Marburg virus (MARV).
Investigational
Remune
Investigational
MVA-mBN226B
Investigational
Rivabazumab pegol
Rivabazumab pegol is being investigated as a treatment of Pseudomonas aeruginosa lung infections in patients with cystic fibrosis.
Investigational
MOR-22
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
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
AVI-7287
Investigational
Hantaan virus DNA vaccine
Investigational
LJP-539
Investigational
Humanized monoclonal antibody Hu1B7
Investigational
ENTR-601-44
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
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
QR-313
QR-313 is an antisense oligonucleotide targeting exon 73. It is being investigated for the treatment of dystrophic epidermolysis bullosa.
Investigational
Displaying drugs 15301 - 15325 of 15564 in total