Gene, genetics and genetic medicines in gastroenterology: Current status and its future

Table 12 Different gene therapy under research/evaluation[150-164]

Therapy/product	Target	Ref.
Alicaforsen (antisense targeting ICAM-1) (phase III)	Pouchitis, left-sided UC	Greuter et al[150]
Glybera (AAV1-LPL) (withdrawn)	Lipoprotein lipase deficiency (severe pancreatitis)	Ferreira et al[151]
Oncolytic AAV-DC-CTL (phase 1)	Stage IV gastric cancer	Yan et al[152]
CRISPRedited TIL therapy (phase 1 completed)	Metastatic GI cancers (colorectal, pancreas, gallbladder, esophagus, stomach)	Lou et al[153]
CTX131 (allogeneic, CRISPR-engineered CD70-CAR-T) (phase 1/2 trial)	Pancreatic/oesophageal cancers	Pal et al[154]
CAN2409 (HSV thymidine kinase gene + pro-drug) (phase 2a)	Pancreatic cancer	Garrett Nichols et al[155]
Mutogene cevumeran (personalized mRNA vaccine) (phase 1b)	Pancreatic ductal adenocarcinoma	Lopez et al[156]
GENEGUT (preclinical settings)	Crohn’s disease	Hoffmann et al[157]
AAVrh.10mAnti-Eos, a serotype rh.10 AAV vector coding for an anti-Siglec-F monoclonal antibody (preclinical)	Eosinophill esophagitis	Camilleri et al[158]
Local delivery of an adenoviral vector expressing the HSV-tk gene (aglatimagene besadenovec, AdV-tk) followed by anti-herpetic prodrug	Pancreatic cancer	Aguilar et al[159]
Thymidine kinase-based gene therapy	HCC	Sangro et al[160]
Adenovirus-mediated double-suicide gene therapy	PDAC	Lee et al[161]
Oncolytic virus pelareorep (reolysin) (phase 1/2 trial)	PDAC	Noonan et al[162]
GVAX pancreas prime and Listeria Monocytogenes expressing mesothelin (CRS-207) boost vaccines (preclinical)	PDAC	Le et al[163]
TNF-erade biologic (phase 1)	Esophageal cancer	Chang et al[164]
GNT-0003 (phase III trial)	Crigler-Najjar syndrome
Pexa-Vec (JX-594) (phase 3 trial)	HCC
DTX401 (AAV8-G6Pase gene therapy) (phase 3 trial)	Glycogen storage disorder 1a
DTX301 (avalotcagene ontaparvovec) (phase 3 trial)	Ornithine transcarbamylase deficiency
UX701 (rivunatpagene miziparvovec) (AAV9) (phase 1/2 trial)	Wilson disease
VTX-802 (preclinical study)	PFIC type 2 (BSEP)

ICAM-1: Intercellular adhesion molecule 1; AAV: Adeno-associated virus; LPL: Lipoprotein lipase; DC: Dendritic cell; CTL: Cytotoxic T lymphocyte; CRISPR: Clustered regularly interspaced short palindromic repeats; TIL: Tumor-infiltrating lymphocyte; CAR T: Chimeric antigen receptor T-cell; CD: Cluster of differentiation; HSV: Herpes simplex virus; mRNA: Messenger RNA; AAVrh.10: Adeno-associated virus serotype rh.10; Siglec-F: Sialic acid-binding immunoglobulin-like lectin F; HSV-tk: Herpes simplex virus thymidine kinase; AdV-tk: Adenoviral vector carrying herpes simplex virus thymidine kinase gene; GVAX: Granulocyte-macrophage colony-stimulating factor secreting tumor cell vaccine; TNF-erade: Tumor necrosis factor-alpha encoding adenoviral vector; UC: Ulcerative colitis; GI: Gastrointestinal; PDAC: Pancreatic ductal adenocarcinoma; HCC: Hepatocellular carcinoma; PFIC: Progressive familial intrahepatic cholestasis; BSEP: Bile salt export pump.