Displaying drugs 14976 - 15000 of 15902 in total
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
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
Matched Description: … mRNA-3927 is an investigational mRNA therapy that consists of two mRNAs that encode for the alpha and …
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
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 …
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
Matched Description: … It binds to an MHC class II molecule and is recognized by a Factor VIII-specific T cell.[L46138] …
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
NM3086
NM3086 is a recombinant, humanized, aglycosylated, IgG1-kappa anti-properdin monoclonal antibody being developed by Novelmed Therapeutics Inc.
Experimental
RLYB212
RLYB212 is a human monoclonal antibody against Antigen-1A Immunoglobulin (HPA-1A). It is currently being developed by Rallybio for the prevention of fetal and neonatal alloimmune thrombocytopenia.
Investigational
Matched Description: … It is currently being developed by Rallybio for the prevention of fetal and neonatal alloimmune thrombocytopenia …
Recombinant Factor XIII (human)
Investigational
Tubimod
Investigational
Staphylococcus aureus immune globulin (human)
Investigational
MCMV5322A
MCMV5322A is an anticytomegalovirus monoclonal IgG1 antibody used in combination with MCMV3068A to form RG7667.
Investigational
BAL200
Investigational
Puumala virus DNA vaccine
Investigational
Humanized monoclonal antibody Hu11E6
Investigational
Displaying drugs 14976 - 15000 of 15902 in total