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Displaying drugs 14801 - 14825 of 15227 in total
Investigational
Investigational
Investigational
Investigational
VE303 is under clinical development by Vedanta Biosciences.
Investigational
Investigational
Investigational
Investigational
Apc001 is an aptamer developed for the treatment of osteogenesis imperfecta.
Investigational
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
Investigational
QR-313 is an antisense oligonucleotide targeting exon 73. It is being investigated for the treatment of dystrophic epidermolysis bullosa.
Investigational
WVE-003 is an allele-selective antisense oligonucleotide that works to selectively reduce the toxic mutant huntingtin (mHTT) protein.
Investigational
NGGT-002 is a recombinant adeno-associated virus serotype 8 vector encoding human phenylalanine hydroxylase (PAH).
Investigational
Investigational
Investigational
Investigational
TOL2 is a modified form of the acetylcholine receptor being investigated for the treatment of myasthenia gravis.
Investigational
Investigational
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 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
Displaying drugs 14801 - 14825 of 15227 in total