Displaying drugs 14951 - 14975 of 15713 in total
mRNA-3351
mRNA-3351 is a modified mRNA encoding the UGT1A1 gene. Developed by Moderna, it is currently being investigated as the treatment for Crigler-Najjar syndrome
Experimental
OMS906
OMS906 is a humanized Igg4 monoclonal antibody against mannan-binding lectin-associated serine protease-3 (MASP-3) currently being developed by Omeros.
Investigational
MCMV3068A
MCMV3068A is an anticytomegalovirus monoclonal IgG1 antibody used in combination with MCMV5322A to form RG7667.
Investigational
VAC-52150
Investigational
RST-001
RST-001 is a non-replicating recombinant adeno-associated virus vector containing a fragment of the gene encoding channel rhodopsin-2 protein.
Investigational
GS030-DP
GS030-DP is an optogenetic gene therapy being investigated for the treatment of retinitis pigmentosa. It targets retinal ganglion cells that encodes a light-sensitive channelrhodopsin, ChrimsonR-tdTomato (ChR-tdT), delivered by an AAV2.7m8 vector. It is a component of GS030, an investigational optogenetic treatment, in combination with a medical device GS030-MD.[A259816, L46701]
Investigational
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
Tonabacase
Investigational
Tabituximab barzuxetan yttrium Y-90
A version of tabituximab barzuxetan radiolabeled with yttrium-90.
Investigational
Displaying drugs 14951 - 14975 of 15713 in total