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Displaying drugs 14351 - 14375 of 15260 in total
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
HMI-103
HMI-102 is an investigational gene therapy designed to encode the phenylalanine-4-hydroxylase (PAH) gene, mutated in people with phenylketonuria.
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
TT 173
TT 173 is a Modified Human Tissue Factor currently being investigated to treat bleeding disorders such as hemophilia and von Willebrand disease, as well as bleeding episodes in cutaneous injuries or dental procedures.
Investigational
Matched Description: … Modified Human Tissue Factor currently being investigated to treat bleeding disorders such as hemophilia and …
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
Tuvirumab
Investigational
Remune
Investigational
Larcaviximab
Investigational
TCN-202
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
Docaravimab
Investigational
Hantaan virus DNA vaccine
Investigational
LJP-539
Investigational
Displaying drugs 14351 - 14375 of 15260 in total