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Displaying drugs 1401 - 1425 of 2248 in total
MYR-101
MYR-101 (rAAV-Olig001-ASPA) is a recombinant adeno-associated virus (rAAV) vector-based gene therapy intended to treat Canavan disease. Canavan disease is a fatal childhood genetic disorder characterized by white matter degeneration in the brain. It is caused by a mutation in the aspartoacylase gene (ASPA), which leads to a deficiency of the...
Investigational
SBT-100
SBT-100 is an anti-signal transducer and activator of transcription 3 (STAT3) B VHH13 single-domain antibody (sdAb). It was granted orphan drug designation by the FDA for the treatment of pancreatic cancer and osteosarcoma.[L44131,L44136]
Investigational
SUN-101
Investigational
OTL-103
Investigational
DYN-101
DYN-101 is an antisense product candidate designed to reduce the expression of dynamin 2 protein.
Investigational
AVS-100
Investigational
DF-1001
Investigational
FBX-101
FBX-101 is an adeno-associated virus serotype rhesus 10 vector expressing the human galactocerebrosidase (GALC) gene
Investigational
Matched Description: … FBX-101 is an adeno-associated virus serotype rhesus 10 vector expressing the human galactocerebrosidase …
TG-1042
TG-1042 (formerly known as ASN-002) is a recombinant adenovirus carrying the cDNA coding sequence for the human interferon gamma gene.
Investigational
CTR-107
CTR-107 is a synthetic targeted growth factor. It is being investigated for the treatment of familial exudative vitreoretinopathy.
Investigational
LMB-100
LMB-100 is a is an antibody-toxin conjugate with an antimesothelin Fab linked to a 24-kilodalton portion of Pseudomonas exotoxin A with mutations that decrease immunogenicity.
Investigational
AVXS-301
AVXS-301 consists of an adeno-associated virus serotype 9 (AAV9) designed to express the superoxide dismutase 1 (SOD1) gene. It is being investigated for the treatment of Amyotrophic Lateral Sclerosis (ALS).
Investigational
Matched Description: … AVXS-301 consists of an adeno-associated virus serotype 9 (AAV9) designed to express the superoxide dismutase …
Lumiflavin
Experimental
LJP 1082
LJP 1082 is a Toleragen that is designed to shut down the B cells that produce antibodies to ß2 GP1. LJP 1082 is undergoing trial for for the treatment of stroke, deep-vein thrombosis and other conditions associated with antibody-mediated thrombosis. Antibody-mediated thrombosis, also called antiphospholipid syndrome (APS), is a blood...
Investigational
NCX 1015
NCX 1015 is nitric oxide-releasing derivative of prednisolone developed for the treatment of inflammatory bowel disease (IBD) by NicOx SA.
Investigational
ETC-1001
ETC-1001 is Pfizer’s lead oral small molecule product candidate which is intended to be a chronic treatment for individuals with lipid disorders.
Investigational
PRTX-100
PRTX-100 is a highly-purified form of Staphylococcal Protein A that binds directly to monocytes and a subset of B-cells that are involved in the development and progression of various autoimmune diseases. These binding interactions enable the compound to modulate the function of these cells and restore the balance of the...
Investigational
CUDC-101
CUDC-101 has been used in trials studying the treatment of Cancer, Tumors, Liver Cancer, Breast Cancer, and Gastric Cancer, among others.
Investigational
CXL-1020
CXL-1020 is under investigation in clinical trial NCT01092325 (Safety and Hemodynamic Effects and Pharmacokinetics of CXL-1020 in Patients With Stable Heart Failure).
Investigational
SXR-1096
Investigational
VLX-1005
Investigational
ATSN-101
ATSN-101 is a adeno-associated virus serotype 5 encoding human retinal guanylate cyclase 1 gene (AAV5-GUCY2D)
Investigational
Displaying drugs 1401 - 1425 of 2248 in total