Displaying drugs 12876 - 12900 of 14392 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
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
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Withdrawn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Withdrawn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Withdrawn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
Matched Description: … construct promoting co-expression of both codon-optimized Poly-A Binding Protein Nuclear-1 (PABPN1) and …
GliSODin
Investigational
Nutraceutical
Darleukin
Investigational
Displaying drugs 12876 - 12900 of 14392 in total