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Displaying drugs 12601 - 12625 of 14373 in total
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
FOV 2501
Investigational
Recombinant human type VII collagen
Investigational
Exon 55 specific phosphorothioate oligonucleotide
Investigational
Human allogeneic bone marrow derived osteoblastic cells
Investigational
Recombinant human galactocerebrosidase
Investigational
Recifercept
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
Human umbilical tissue derived cells
Investigational
CEQ508
CEQ508 is a Live Attenuated E. Coli Expressing Beta Catenin ShRNA currently being investigated to treat Familial Adenomatous Polyposis.
Investigational
pGM169/GL67A
Investigational
Engensis
Investigational
IR-901
Investigational
Lexanersen
Lexanersen is an antisense oligonucleotide under investigation for use as a disease-modifying therapy in Huntington's disease.
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
Human ciliary neurotrophic factor, recombinant (E. coli)
Investigational
Displaying drugs 12601 - 12625 of 14373 in total