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Displaying drugs 14901 - 14925 of 15636 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

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

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