Displaying drugs 11501 - 11525 of 12787 in total
ARO-APOC3
ARO-APOC3 is an investigational RNAi therapeutic targeting apolipoprotein C-III (APOC3). It received an Orphan Drug designation by the FDA for the treatment of familial chylomicronemia syndrome.
Investigational
Tividenofusp alfa
Tividenofusp alfa, also known as DNL-310, is a brain-penetrant enzyme replacement therapy being investigated for the treatment of Hunter syndrome.
Investigational
INZ-701
INZ-701 is an enzyme replacement therapy under development with the intention to be used for the treatment of calcification disorders of the circulatory system, bones, and kidneys.
Investigational
RGX-111
RGX-111 is an adeno-associated virus vector AAV9 designed to deliver the α-L-iduronidase (IDUA) gene. Developed by REGENXBIO Inc., it was investigated for the treatment of mucopolysaccharidosis Type I (MPS I).
Investigational
AAV2/8.TBG.hARSB
Investigational
JLK-247
JLK-247 is an adeno-associated viral vector serotype 9 (AAV9)-mediated gene replacement therapy for the treatment of Sanfilippo Syndrome Type C or MPS IIIC.
Investigational
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
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
EGT-301
EGT-301 is a gene therapy being investigated for Hunter Syndrome. It consists of an adeno-associated viral vector serotype 9 (AAV9) containing the human Iduronate-2-sulfatase (I2S) transgene designed to restore I2S functional deficiency in patients with Hunter syndrome.
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
mRNA-3927
mRNA-3927 is an investigational mRNA therapy that consists of two mRNAs that encode for the alpha and beta subunits of the mitochondrial enzyme propionyl-CoA carboxylase (PCC) encapsulated within a lipid nanoparticle (LNP). Developed by Moderna Inc., it is being investigated for the treatment of propionic acidemia.
Investigational
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
GTP-506
GTP-506 is a gene therapy formed by two vectors, a nuclease vector targeting gene editing in the well-characterized PCSK9 gene locus and a therapeutic donor vector that inserts the OTC gene to provide the desired genetic correction.
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
WP-1301
WP1301 is a synthetic peptide derived from hFVIII. It binds to an MHC class II molecule and is recognized by a Factor VIII-specific T cell.
Investigational
Displaying drugs 11501 - 11525 of 12787 in total