Displaying drugs 13976 - 14000 of 14690 in total
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
BMN-045
Experimental
Withdrawn
AMZ002
AMZ002 is a sterile injectable hormone developed as a purified synthetic polypeptide alternative to current FDA-approved epileptic seizure treatments.
Investigational
Displaying drugs 13976 - 14000 of 14690 in total