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Displaying drugs 13451 - 13475 of 14350 in total

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