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Displaying drugs 13276 - 13300 of 14353 in total
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 …
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
PRX-ASL
PRX-ASL is an intracellular enzyme replacement therapy designed to replace the missing or defective enzyme in patients with argininosuccinate lyase deficiency.
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
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
NM3086
NM3086 is a recombinant, humanized, aglycosylated, IgG1-kappa anti-properdin monoclonal antibody being developed by Novelmed Therapeutics Inc.
Experimental
Valziflocept
Investigational
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
Zamerovimab
Investigational
MBL-HCV1
MBL-HCV1 is a monoclonal antibody developed to prevent the recurrence of hepatitis C virus (HCV) in patients receiving a liver transplant.
Investigational
Recombinant soluble human CD4
Investigational
Displaying drugs 13276 - 13300 of 14353 in total