Displaying drugs 13426 - 13450 of 14392 in total
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
PTR-01
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
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
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
CNM-Au8
Investigational
Human Embryonic Stem Cell-derived Astrocytes
Investigational
GALE-302
Investigational
C-Alpha-stx2
Investigational
Viralym-C
Viralym-C is an investigational T-cell immunotherapy developed by ViraCyte. It consists of Human Leukocyte Antigen (HLA)-matched cytomegalovirus (CMV)-specific T lymphocytes. It was granted FDA orphan designation in January 2017 and investigated for the treatment of CMV infection.[A260291, L47241]
Investigational
Matched Description: … It was granted FDA orphan designation in January 2017 and investigated for the treatment of CMV infection …
ISIS-333611
Investigational
Recombinant human palmitoyl protein thioesterase-1
Investigational
Baloramotide
Investigational
Displaying drugs 13426 - 13450 of 14392 in total