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Displaying drugs 13376 - 13400 of 14392 in total
Ranuzifigene civaparvovec
Experimental
Adlinacogene civaparvovec
Adlinacogene civaparvovec is an investigational in vivo genome editing therapeutic product. It uses proprietary zinc finger nuclease (ZFN) genome editing to insert into the DNA of liver cells a copy of the F9 gene, which controls the production of Factor IX. Developed by Sangamo Therapeutics Inc., it is being investigated...
Experimental
Devafidugene civaparvovec
Devafidugene civaparvovec is an investigational in vivo genome editing product. It uses proprietary zinc finger nucleases (ZFN) that insert a functional copy of the alpha-L-iduronidase (IDUA) enzyme. Developed by Sangamo Therapeutics, Inc., devafidugene civaparvovec is being investigated for the treatment of mucopolysaccharidosis Type I (MPS I).
Experimental
Dirloctocogene samoparvovec
Investigational
Cotoretigene toliparvovec
Investigational
Entacingene turiparvovec
Investigational
Fordadistrogene movaparvovec
Investigational
Pariglasgene brecaparvovec
Investigational
Patidistrogene bexoparvovec
Investigational
Domofenogene zalfaparvovec
Domofenogene zalfaparvovec, formerly known as BMN-307, is an investigational AAV5-phenylalanine hydroxylase (PAH) gene therapy developed by BioMarin Pharmaceutical.
Experimental
Ifezuntirgene inilparvovec
Experimental
Ixoberogene soroparvovec
Investigational
Zaftuclenegene piruparvovec
Experimental
Human Laminin-111
Investigational
Recombinant methionyl brain-derived neurotrophic factor
Investigational
Tegoprubart
Investigational
ABO-202
Investigational
Spheramine
Investigational
BBP-812
Investigational
TSHA-120
Investigational
TVN-102
Investigational
Vesleteplirsen
Investigational
CV-MG01
Investigational
AT-GTX-502
AT-GTX-502 is an experimental, one-time gene therapy that uses AAV9 to deliver a functional copy of CLN3.
Investigational
Displaying drugs 13376 - 13400 of 14392 in total