Displaying drugs 11376 - 11400 of 12789 in total
Lanacogene vosiparvovec
Experimental
Volrubigene ralaparvovec
Volrubigene ralaparvovec is under investigation in clinical trial NCT03223194 (Gene Transfer Clinical Study in Crigler-najjar Syndrome).
Investigational
Tefidsogene civaparvovec
Tefidsogene civaparvovec, formerly known as SB-913, is an investigational therapy for genome editing. It consists of an adeno-associated virus (AAV) type 2/6 vector and a zinc finger nuclease (ZFN) system that inserts a correct copy of the Iduronate 2-Sulfatase (IDS) gene into the albumin locus in hepatocytes. Developed by Sangamo...
Experimental
Zildistrogene varoparvovec
Experimental
Ezaladcigene resoparvovec
Investigational
Avalotcagene ontaparvovec
Investigational
Cobnabexagene anvuparvovec
Experimental
Bomtabegagene bavoparvovec
Investigational
Verbrinacogene setparvovec
Investigational
Botaretigene sparoparvovec
Investigational
Engabexagene cincesparvovec
Experimental
NeuroVax
Investigational
GL-2045
Investigational
AGIL-FA
Investigational
Latozinemab
Investigational
GTX-102
GTX-102 is an antisense oligonucleotide designed to target and inhibit the expression of UBE3A-AS. It is being investigated for the treatment of Angelman Syndrome.
Investigational
BLS-M22
Investigational
TSHA-118
TSHA-118 is a self-complementary AAV9 gene replacement therapy designed to express a human codon-optimized CLN1 transgene for the treatment of CLN1 disease.
Experimental
PR006
PR006 is a non-replicating recombinant adeno-associated virus serotype 9 containing the progranulin gene. It is being investigated for the treatment of dementia.
Investigational
BMN-044
Experimental
Withdrawn
BMN-046
Experimental
Withdrawn
Alirinetide
Investigational
BMN-055
Experimental
Withdrawn
CTI-1601
Investigational
BB-301
BB-301 is a modified AAV serotype 9 (AAV9) capsid expressing a unique, single bifunctional construct promoting co-expression of both codon-optimized Poly-A Binding Protein Nuclear-1 (PABPN1) and two small inhibitory RNAs (siRNAs) against mutant PABPN1. Developed by Benitec Biopharma, BB-301 is being investigated for the treatment of Oculopharyngeal Muscular Dystrophy (OPMD).
Investigational
Displaying drugs 11376 - 11400 of 12789 in total