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Displaying drugs 14626 - 14650 of 15088 in total
Luveltamab tazevibulin
Luveltamab tazevibulin, formerly known as STRO-002, is novel homogeneous folate receptor alpha (FolRα) targeting antibody-drug conjugate. It is under investigation in clinical trial NCT05200364 (A Study of STRO-002, an Anti-folate Receptor Alpha Antibody Drug Conjugate, in Combination With Bevacizumab in Epithelial Ovarian Cancer).
Investigational
DCVax-L
DCVax-L is a personalized immunotherapy comprising an autologous vaccine with dendritic cells loaded in vitro with autologous tumour lysate.
Investigational
Strimvelis
Investigational
EI-001
Investigational
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
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
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
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
Displaying drugs 14626 - 14650 of 15088 in total