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Displaying drugs 13276 - 13300 of 14353 in total
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 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
Investigational
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 is designed to use the NAV AAV9 vector to deliver the human iduronate-2-sulfatase gene to the central nervous system.
Investigational
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
Investigational
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 is a CRISPR-edited allogeneic CAR-T cell therapy targeting CD19. It is being investigated for the treatment of follicular lymphoma.
Investigational
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 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 is a long-acting carboxy-terminal peptide (CTP)-modified Factor IIa. It is being investigated for the treatment of hemophilia.
Investigational
Experimental
NM3086 is a recombinant, humanized, aglycosylated, IgG1-kappa anti-properdin monoclonal antibody being developed by Novelmed Therapeutics Inc.
Experimental
Investigational
Investigational
MCMV5322A is an anticytomegalovirus monoclonal IgG1 antibody used in combination with MCMV3068A to form RG7667.
Investigational
Investigational
MBL-HCV1 is a monoclonal antibody developed to prevent the recurrence of hepatitis C virus (HCV) in patients receiving a liver transplant.
Investigational
Displaying drugs 13276 - 13300 of 14353 in total