Displaying drugs 14776 - 14800 of 15841 in total
WVE-N531
Investigational
OCU-100
Investigational
SPL23-2
SPL23-2 is an antisense oligonucleotide (ASO) targeting the W1282X nonsense mutation associated with a severe form of cystic fibrosis.
Investigational
QR-1123
QR-1123 is a first-in-class investigational RNA-based oligonucleotide designed to treat autosomal dominant retinitis pigmentosa due to the P23H mutation in the rhodopsin (RHO) gene.
Investigational
AAV-dXIAP
AAV-dXIAP is a recombinant adeno-associated virus-encoded gene for X-linked mammalian inhibitor of apoptosis protein (XIAP) developed by Neurologix.
Investigational
Exon 55 specific phosphorothioate oligonucleotide
Investigational
Recombinant human galactocerebrosidase
Investigational
Apc001PE
Investigational
Haptoglobulin (human)
Investigational
GIVI-MPC
GIVI-MPC is a therapy that uses human-induced pluripotent stem cell-derived myogenic progenitor cells.
Investigational
pGM169/GL67A
Investigational
Engensis
Investigational
OPC1
OPC1 is an oligodendrocyte progenitor cell therapy being investigated for the treatment of acute spinal cord injury.
Investigational
AP-101
AP-101 is a recombinant human antibody directed against human misfolded superoxide dismutase 1.
Investigational
OT-15
Investigational
ABO-201
Investigational
NSI-566
NSI-566 is a stem cell therapy consisting of human spinal cord-derived neural stem cells. Developed by Seneca Biopharma, it is being investigated for the treatment of amyotrophic lateral sclerosis (ALS), stroke, and chronic spinal cord injury (cSCI).
Investigational
Matched Description: … Biopharma, it is being investigated for the treatment of amyotrophic lateral sclerosis (ALS), stroke, and …
Tonabacase
Investigational
Tabituximab barzuxetan yttrium Y-90
A version of tabituximab barzuxetan radiolabeled with yttrium-90.
Investigational
Yttrium Y-90 Labetuzumab
Investigational
BHT-3034
BHT-3034 is a DNA plasmid vaccine immunotherapy under investigation for the treatment of myasthenia gravis.
Investigational
SIS-101-ADO
SIS-101-ADO is a siRNA therapeutic under investigation for the treatment of Autosomal Dominant Osteopetrosis Type 2 (ADO2).
Investigational
PBKR03
PBKR03 is a non-replicating recombinant adeno-associated virus serotype hu68 vector, containing a transgene encoding the human galactosylceramidase (GALC) enzyme.
Investigational
ATX-01
ATX-01 is an antimiR, a chemically modified antisense oligonucleotide, designed to target microRNA 23b (miR-23b).
Investigational
Displaying drugs 14776 - 14800 of 15841 in total