Displaying drugs 14626 - 14650 of 15206 in total
Hantaan virus DNA vaccine
Investigational
LJP-539
Investigational
Humanized monoclonal antibody Hu1B7
Investigational
ENTR-601-44
ENTR-601-44 is an exon 44 skipping phosphorodiamidate morpholino oligonucleotide (PMO). Developed by Entrada Therapeutics, it was investigated for the treatment of Duchenne muscular dystrophy.
Investigational
Sonpiretigene isteparvovec
Sonpiretigene isteparvovec is an ambient-light activatable Multi-Characteristic Opsin (MCO) optogenetic therapy. Developed by Nanoscope Therapeutics, it is being investigated for vision restoration in advanced retinitis pigmentosa (RP). Sonpiretigene isteparvovec consists of a fusion Adeno-associated virus (AAV) vector with three plasmids encoding opsins with spectrally-separated activation peaks.
Investigational
rsATP7A cDNA
Investigational
AAV-AIPL
AAV-AIPL is an adenovirus-associated viral vector serotype 8 containing the human AIPL1 gene. Developed by MeiraGTx, it is being investigated for the treatment of inherited retinal dystrophy due to defects in AIPL1 gene.
Investigational
QR-313
QR-313 is an antisense oligonucleotide targeting exon 73. It is being investigated for the treatment of dystrophic epidermolysis bullosa.
Investigational
WVE-003
WVE-003 is an allele-selective antisense oligonucleotide that works to selectively reduce the toxic mutant huntingtin (mHTT) protein.
Investigational
NGGT-002
NGGT-002 is a recombinant adeno-associated virus serotype 8 vector encoding human phenylalanine hydroxylase (PAH).
Investigational
PTR-01
Investigational
rAAVrh74.MCK.GALGT2
Investigational
Nomlabofusp
Investigational
TOL2
TOL2 is a modified form of the acetylcholine receptor being investigated for the treatment of myasthenia gravis.
Investigational
Autologous adipose derived mesenchymal stromal cells
Investigational
Autologous cord blood
Investigational
INT41
INT41 (also known as rAAV6-INT41) is a recombinant adeno-associated virus vector containing DNA encoding INT41 intrabody.
Investigational
AVXS-301
AVXS-301 consists of an adeno-associated virus serotype 9 (AAV9) designed to express the superoxide dismutase 1 (SOD1) gene. It is being investigated for the treatment of Amyotrophic Lateral Sclerosis (ALS).
Investigational
HER-902
Investigational
CNM-Au8
Investigational
Human Embryonic Stem Cell-derived Astrocytes
Investigational
Palucorcel
Palucorcel is an investigational stem cell therapy consisting of human umbilical tissue-derived cells. Developed by Janssen Biotech, it is being investigated for the treatment of various conditions such as amyotrophic lateral sclerosis (ALS) and macular degeneration.
Investigational
Matched Description: … being investigated for the treatment of various conditions such as amyotrophic lateral sclerosis (ALS) and …
RPI-MN
RPI-MN is a detoxified or chemically modified alpha-cobratoxin with antiviral properties. Being developed by Nutra Pharma under the trade name PEPTERON, it is being investigated for the treatment of neurological and immune disorders.
Investigational
Matched Description: … Nutra Pharma under the trade name PEPTERON, it is being investigated for the treatment of neurological and …
Displaying drugs 14626 - 14650 of 15206 in total