Advanced Filter

Filter by Group

Filter by Market Availability

Displaying drugs 12801 - 12825 of 14390 in total
Investigational
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 is a non-replicating recombinant adeno-associated virus serotype 9 containing the progranulin gene. It is being investigated for the treatment of dementia.
Investigational
Experimental
Withdrawn
Experimental
Withdrawn
Investigational
Experimental
Withdrawn
Investigational
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
Investigational
Nutraceutical
Investigational
Investigational
Investigational
Investigational
Investigational
Investigational
Investigational
Investigational
Investigational
Investigational
Investigational
MGD-024 is an anti-CD123/anti-CD3 bispecific DART molecule.
Investigational
ST-36 is a cell-penetrating dominant-negative form of activating transcription factor 5. In 2017, it received orphan drug designation by the FDA for the treatment of glioma.
Investigational
IMAB027 is a chimeric monoclonal antibody against Claudin 6.
Investigational
LV305 is a lentiviral vector encoding NY-ESO-1 gene. LV305 is an active component of CMB305 along with G305.
Investigational
Displaying drugs 12801 - 12825 of 14390 in total