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Displaying drugs 12976 - 13000 of 14388 in total
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
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
AVR-RD-01
AVR-RD-01 is an investigational ex vivo lentiviral gene therapy developed by AVROBIO Inc. It consists of autologous CD34+ cells that transduced with Lentiviral Vector/alpha-galactosidase A (AGA) encoding for the human AGA complementary deoxyribonucleic acid (cDNA) sequence. AVR-RD-01 is being investigated for Fabry Disease.[L45963, L45968]
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
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
Displaying drugs 12976 - 13000 of 14388 in total