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Displaying drugs 26 - 42 of 42 in total
CTX110 is an allogeneic CRISPR/Cas9-mediated genetically modified CAR T cell therapy targeting CD19 antigen.
Investigational
CTX130 is an allogeneic CRISPR/Cas9-mediated genetically modified CAR T cell therapy targeting CD70 antigen.
Investigational
Investigational
CTX120 is an allogeneic CRISPR/Cas9-mediated genetically modified CAR T cell therapy targeting B-cell maturation antigen.
Investigational
Experimental
VOR33 is an allogeneic CRISPR/Cas9 genome-edited hematopoietic stem and progenitor cell (HSPC) therapy product lacking the CD33 protein.
Investigational
NTLA-2002 is a lipid nanoparticle encapsulating single guide RNA (G012267) targeting the human KLKB1 gene and messenger RNA (mRNA000042) encoding Cas9. It is an investigational therapy designed to knock out the target gene kallikrein B1 (KLKB1) to reduce plasma kallikrein activity, thereby preventing hereditary angioedema attacks.
Investigational
EDIT-101 is a recombinant adeno-associated virus pseudotyped with serotype 5 viral capsid encoding CRISPR/Cas9 (clustered regularly interspaced short palindromic repeats associated protein 9) gene editing constructs.
Investigational
CAStem is composed of immunity- and matrix-regulatory cells (IMRCs), also known as M cells, which can be administered through injection. These IMRCs are differentiated from human embryonic stem cells that are expanded and then harvested. These cells are currently being investigated against lung injury and fibrosis -- conditions which are...
Investigational
TN-401 is a native AAV9 capsid with a genomic cassette containing a cardiomyocyte-specific promoter and the wild-type human PKP2 gene.
Investigational
TN-201 is a gene therapy comprising a native AAV9 capsid with a genomic cassette containing a cardiomyocyte-specific promoter and the wild-type human MYBPC3 gene.
Investigational
Investigational
Tefidsogene civaparvovec, formerly known as SB-913, is an investigational therapy for genome editing. It consists of an adeno-associated virus (AAV) type 2/6 vector and a zinc finger nuclease (ZFN) system that inserts a correct copy of the Iduronate 2-Sulfatase (IDS) gene into the albumin locus in hepatocytes. Developed by Sangamo...
Experimental
Investigational
Suratadenoturev is a gene-modified oncolytic adenovirus expressing the telomerase reverse transcriptase gene.
Investigational
Displaying drugs 26 - 42 of 42 in total