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Displaying drugs 13051 - 13075 of 14390 in total
PA3-17 is CD7-directed chimeric antigen receptor (CAR) T-cell therapy.
Investigational
ETX101 is a non-replicating recombinant adeno associated viral vector, serotype 9, designed to promote increased transcription of SCN1A gene.
Investigational
PBGM01 is a non-replicating recombinant adeno-associated virus serotype hu68 vector, which contains human GLB1 transgene encoding the galactosidase beta 1 enzyme.
Investigational
CTR-107 is a synthetic targeted growth factor. It is being investigated for the treatment of familial exudative vitreoretinopathy.
Investigational
M1-c6v1 is a recombinant oncolytic virus.
Investigational
TSHA-102 is a recombinant serotype 9 adeno-associated virus encoding a codon-optimized human MECP2 gene.
Investigational
Perenostobart is an alpha-CD39 IgG4 monoclonal antibody.
Investigational
ECT204 is an autologous T-cell therapy whereby T cells are genetically modified to target tumor-specific antigens.
Investigational
Sapablursen is a synthetic 2'-O-(2-methoxyethyl)-modified antisense oligonucleotide linked to a tri-antennary cluster of N-acetyl galactosamine (GalNAc3) sugars targeting transmembrane protease, serine 6 messenger ribonucleic acid
Investigational
ST266 is an amniotic membrane-derived cellular cytokine solution.
Investigational
VGB-R04 is an adeno-associated viral vector delivering human FIX Padua gene.
Investigational
CTX130 is an allogeneic CRISPR/Cas9-mediated genetically modified CAR T cell therapy targeting CD70 antigen.
Investigational
AVR-RD-05 is a gene therapy comprising genetically modified autologous stem cells transduced ex vivo with a lentiviral vector encoding the human iduronate-2-sulfatase (IDS) enzyme. It is under investigation for the treatment of Hunter Syndrome (Mucopolysaccharidosis Type II, MPSII).
Investigational
MaaT013 is an allogeneic fecal microbiota therapy. It is being investigated for the treatment of Graft-Versus-Host Disease.
Investigational
ATIR101 is a haploidentical, naïve cell-enriched T-cell therapy.
Investigational
ssCART-19 cells consist of autologous anti-CD19 Chimeric Antigen Receptors (CAR)-T cells expressing a short hairpin RNA (shRNA) against interleukin-6 (IL-6).
Investigational
Avoplacel (PLX-R18) is an allogeneic cell therapy under investigation for the treatment of acute radiation syndrome.
Investigational
Holoclar (ex vivo expanded autologous human corneal epithelial cells containing stem cells) is a stem cell therapy used to treat limbal stem cell deficiencies.
Investigational
Rugonersen is a locked nucleic acid antisense oligonucleotide under investigation for the treatment of Angelman Syndrome.
Investigational
Displaying drugs 13051 - 13075 of 14390 in total