Displaying drugs 14701 - 14725 of 15637 in total
Lademirsen
Investigational
ARO-ANG3
ARO-ANG3 is a subcutaneously administered RNA interference (RNAi)-based investigational medicine targeting angiopoietin-like protein 3 (ANGPTL3). It was granted an orphan drug designation for the treatment of homozygous familial hypercholesterolemia (HoFH).
Investigational
AT845
Investigational
RGX-501
RGX-501 is an adeno-associated virus vector 8 (AAV8)-mediated gene therapy that delivers a functional copy of the human low-density lipoprotein receptor (LDLR) gene to liver cells. It is being investigated for the treatment of homozygous familial hypercholesterolemia.
Investigational
EGT-101
EGT-101 is an adeno-associated viral vector serotype 9 (AAV9) vector-based gene therapy consisting of the human sulfamidase (SGSH) transgene. Developed by Esteve Pharmaceuticals, it is being investigated for Sanfilippo syndrome type A.
Investigational
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
ACTUS-101
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
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
Displaying drugs 14701 - 14725 of 15637 in total