Displaying drugs 15251 - 15275 of 15530 in total
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
GALE-302
Investigational
ISIS-333611
Investigational
Recombinant human palmitoyl protein thioesterase-1
Investigational
SGN-1
SGN-1 is a genetically modified strain of Salmonella enterica comprising methioninase.
Investigational
Alidornase alfa
Investigational
Eneboparatide
Investigational
Haptoglobulin 1/1 (Plasma Derived)
Investigational
KB407
Investigational
Belcesiran
Investigational
SPK-7001
Investigational
M012
Investigational
Nadunolimab
Investigational
OAV401
Investigational
ET-101
Investigational
Displaying drugs 15251 - 15275 of 15530 in total