Displaying drugs 14076 - 14100 of 14717 in total
GC5107
GC5107 is an investigational intravenous immunoglobulin. It is marketed as IVIG-SN.
Investigational
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
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
HMI-103
HMI-102 is an investigational gene therapy designed to encode the phenylalanine-4-hydroxylase (PAH) gene, mutated in people with phenylketonuria.
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
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
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
Displaying drugs 14076 - 14100 of 14717 in total