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Displaying drugs 14901 - 14925 of 15902 in total
Verbrinacogene setparvovec
Investigational
Botaretigene sparoparvovec
Investigational
NeuroVax
Investigational
GL-2045
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Withdrawn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Withdrawn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Withdrawn
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 …
MTI-201
Investigational
OS2966
OS2966 is a humanized integrin β1 (CD29) blocking antibody currently evaluated for the treatment of acute myeloid leukemia, glioblastoma and ovarian cancer.[A254561, L47351]
Investigational
Matched Description: … CD29) blocking antibody currently evaluated for the treatment of acute myeloid leukemia, glioblastoma and …
Murlentamab
Investigational
Lerapolturev
Investigational
ALT-P7
Investigational
ST-36
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
LV305
LV305 is a lentiviral vector encoding NY-ESO-1 gene. LV305 is an active component of CMB305 along with G305.
Investigational
SNS01-T
SNS01-T is a gene therapy nanoparticle. It is composed of three components: a B cell-specific plasmid expressing eIF5AK50R; an siRNA that targets the native eIF5A that promotes growth/anti-apoptosis of cancer cells; and polyethylenimine.
Investigational
Matched Description: … eIF5AK50R; an siRNA that targets the native eIF5A that promotes growth/anti-apoptosis of cancer cells; and …
Inalimarev
Investigational
Displaying drugs 14901 - 14925 of 15902 in total