Displaying drugs 15476 - 15500 of 16219 in total
AEM-28-14
AEM-28-14 is an analog of AEM-28, an apolipoprotein E mimetic peptide, with a 400 percent greater cholesterol-lowering efficacy and a several-fold increase in drug tolerability. It is being investigated for lipid disorders such as homozygous familial hypercholesterolemia.
Investigational
Matched Description: … -28], an apolipoprotein E mimetic peptide, with a 400 percent greater cholesterol-lowering efficacy and …
RP-A501
RP-A501 is a gene therapy product consisting of a recombinant adeno-associated virus serotype 9 (rAAV9) capsid containing the human LAMP2B transgene (AAV9:LAMP2B), which encodes lysosome-associated membrane protein 2 (LAMP-2). It is being investigated for Danon disease.
Investigational
OTL-201
OTL-201 is an investigational hematopoietic stem cell (HSC) gene therapy being developed by Orchard Therapeutics for the treatment of mucopolysaccharidosis type IIIA (MPS-IIIA). It consists of a lentiviral vector to insert a functional copy of the human N-Sulfoglucosamine Sulfohydrolase (SGSH) gene into autologous CD34+ hematopoietic stem cells.
Investigational
Reloxiliase
Reloxiliase is a first-in-class, oral crystalline formulation of the oxalate-specific, microbial enzyme oxalate decarboxylase. It is being investigated for the treatment of hyperoxaluria.
Investigational
MRT5201
Investigational
DCR-PH1
Investigational
SYNB1020
SYNB1020 is an engineered strain of Escherichia coli Nissle 1917 developed by Synlogic Inc. It was initially designed to convert ammonia to L-arginine and granted orphan designation by the FDA for the treatment of urea cycle disorders in August 2016; however, the manufacturer discontinued further development of SYNB1020 to treat...
Investigational
Matched Description: … It was initially designed to convert ammonia to L-arginine [A258838] and granted orphan designation by …
Recombinant human arylsulfatase A
Investigational
Porphobilinogen deaminase, human recombinant
Investigational
Oxabact
Investigational
RGX-121
RGX-121 is designed to use the NAV AAV9 vector to deliver the human iduronate-2-sulfatase gene to the central nervous system.
Investigational
LB-001
LB-001 is a gene editing therapy designed to incorporate a functioning version of the faulty methylmalonyl-COA mutase (MUT) gene into the genome of patients with methylmalonic acidemia.
Investigational
AGT-184
Investigational
DTX-101
DTX-101 is an investigational gene therapy consisting of an adeno-associated virus serotype Rh10 vector encoding the human Factor Ix gene. It was previously investigated for the treatment of hemophilia B; however, the clinical development of DTX-101 was discontinued.
Investigational
BBM-H803
BBM-H803 is a recombinant adeno-associated virus (AAV) based gene therapy drug for hemophilia A developed and manufactured by Belief BioMed. It consists of an adeno-associated virus vector containing an expression cassette of the human Factor VIII transgene.
Investigational
Matched Description: … BBM-H803 is a recombinant adeno-associated virus (AAV) based gene therapy drug for hemophilia A developed and …
Coagulin-B
Coagulin-B is an adeno-associated viral vector expressing human blood-coagulation Factor IX.[A259038, A259043] It was previously investigated for the treatment of hemophilia; however, the manufacturing company discontinued the Phase I trial in 2004, halting its further development and investigation.
Investigational
Matched Description: … , the manufacturing company discontinued the Phase I trial in 2004, halting its further development and …
CB-010
CB-010 is a CRISPR-edited allogeneic CAR-T cell therapy targeting CD19. It is being investigated for the treatment of follicular lymphoma.
Investigational
MaxAdFVIII
MaxAdFVIII is an adenoviral vector that expresses the full-length human Factor VIII (hFVIII) cDNA. First developed by GenStar, it is being investigated for the treatment of hemophilia.
Investigational
Thalagen
Thalagen is a branded product developed by San Rocco Therapeutics. It consists of autologous CD34-positive hematopoietic progenitor cells transduced with TNS9.3.55, a lentiviral vector encoding the normal human beta-globin gene. It is being investigated for the treatment of beta-thalassemia.
Investigational
MOD-5014
MOD-5014 is a long-acting carboxy-terminal peptide (CTP)-modified Factor IIa. It is being investigated for the treatment of hemophilia.
Investigational
Vensobafusp alfa
Experimental
Valziflocept
Investigational
Recombinant Factor XIII (human)
Investigational
Tubimod
Investigational
Staphylococcus aureus immune globulin (human)
Investigational
Displaying drugs 15476 - 15500 of 16219 in total