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Displaying drugs 13651 - 13675 of 14784 in total
AVI-7287
Investigational
Docaravimab
Investigational
Hantaan virus DNA vaccine
Investigational
LJP-539
Investigational
Apc001
Apc001 is an aptamer developed for the treatment of osteogenesis imperfecta.
Investigational
Matched Description: … Apc001 is an aptamer developed for the treatment of osteogenesis imperfecta. …
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
Matched Description: … Developed by Entrada Therapeutics, it was investigated for the treatment of Duchenne muscular dystrophy …
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
Matched Description: … [L46676] Sonpiretigene isteparvovec consists of a fusion Adeno-associated virus (AAV) vector with three …
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
Matched Description: … Developed by MeiraGTx, it is being investigated for the treatment of inherited retinal dystrophy due …
QR-313
QR-313 is an antisense oligonucleotide targeting exon 73. It is being investigated for the treatment of dystrophic epidermolysis bullosa.
Investigational
Matched Description: … It is being investigated for the treatment of dystrophic epidermolysis bullosa.[A259867] …
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
Matched Description: … It addresses the underlying cause of Usher syndrome due to the c.7595-2144A>G mutation in the _USH2A_ ... first-in-class RNA-based oligonucleotide designed to restore wild-type USH2A mRNA, leading to the production of …
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
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
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
Matched Description: … RGX-181 consists of an adeno-associated virus serotype 9 (AAV9) vector designed to deliver the soluble ... Developed by REGENXBIO, it is being investigated for the treatment of late-infantile neuronal ceroid …
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
Matched Description: … It is being investigated for the treatment of Amyotrophic Lateral Sclerosis (ALS).[L47077] ... AVXS-301 consists of an adeno-associated virus serotype 9 (AAV9) designed to express the superoxide dismutase …
HER-902
Investigational
CNM-Au8
Investigational
Displaying drugs 13651 - 13675 of 14784 in total