Displaying drugs 11376 - 11400 of 12761 in total
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Withdrawn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Withdrawn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Withdrawn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
GliSODin
Investigational
Nutraceutical
Tabelecleucel
Tabelecleucel is an innovative therapy that uses Epstein-Barr virus (EBV)-specific allogeneic cytotoxic T cells (CTLs). It is produced by mixing T-cells with B-cells that have been infected with the Epstein-Barr virus (EBV). Both T-cells and B-cells are obtained from the same donor, and T-cells are grown to increase their numbers....
Investigational
MYR-101
MYR-101 (rAAV-Olig001-ASPA) is a recombinant adeno-associated virus (rAAV) vector-based gene therapy intended to treat Canavan disease. Canavan disease is a fatal childhood genetic disorder characterized by white matter degeneration in the brain. It is caused by a mutation in the aspartoacylase gene (ASPA), which leads to a deficiency of the...
Investigational
Rosomidnar
Investigational
Darleukin
Investigational
MTI-201
Investigational
Algenpantucel-L
Investigational
GTA002
Investigational
CE-VST01-JC
Investigational
KITE-222
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
DP-303c
Investigational
Murlentamab
Investigational
Displaying drugs 11376 - 11400 of 12761 in total