Displaying drugs 13876 - 13900 of 14532 in total
MOR-22
MOR-22, a Natural human lymphoblastoid interferon-alpha, is currently being investigated to treat papillomavirus warts in the oral cavity of HIV-positive patients.
Investigational
INM004
INM004, a Neutralizing Equine Anti-Stx Hyperimmune Immunoglobulin F(Ab')2 Fragment, is currently being investigated to treat Shiga-toxin producing bacterial infection relating to the prevention of hemolytic uremic syndrome.
Investigational
AVI-7287
Investigational
Docaravimab
Investigational
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
QRX-411
QRX-411 is a first-in-class RNA-based oligonucleotide designed to restore wild-type USH2A mRNA, leading to the production of the functional USH2A protein. It addresses the underlying cause of Usher syndrome due to the c.7595-2144A>G mutation in the USH2A gene.
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
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
INT41
INT41 (also known as rAAV6-INT41) is a recombinant adeno-associated virus vector containing DNA encoding INT41 intrabody.
Investigational
RGX-181
RGX-181 consists of an adeno-associated virus serotype 9 (AAV9) vector designed to deliver the soluble lysosomal enzyme tripeptidyl peptidase I (TPP1) gene. Developed by REGENXBIO, it is being investigated for the treatment of late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease.
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
Displaying drugs 13876 - 13900 of 14532 in total