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Displaying drugs 15376 - 15400 of 15597 in total

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

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

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