A review of animal models utilized in preclinical studies of approved gene therapy products: trends and insights

Soufizadeh, Parham; Mansouri, Vahid; Ahmadbeigi, Naser

doi:10.1186/s42826-024-00195-6

Laboratory Animal Research

Table 2 Animal models utilized in preclinical studies of cancer-related products

From: A review of animal models utilized in preclinical studies of approved gene therapy products: trends and insights

Year of Approval	Trade name (General name)	Target cell (in vivo/ex-vivo	Indication	Animal model	Details	Comments	Category	References
2003	Gendicine	In vivo	Head and neck cancer (SCC)	mice	knockout	Conditional knockout mouse model	Genetically Engineered	[25, 26]
2005	Oncorine	In vivo	Nasopharyngeal carcinoma	guinea pigs	N/A	Injected with Oncorine at dose levels of 5.0 × 10¹⁰ TCID50/kg, 1.0 × 10¹¹ TCID50/kg, or 2.0 × 10¹¹ TCID50/kg subcutaneously	N/A	[27]
2005	Oncorine	In vivo	Nasopharyngeal carcinoma	mice	Immunodeficient	Nude—A dose dependent manner from 5 × 10⁴ to 4 × 10⁷ TCID50/mm³	N/A	[27]
2007	Rexin-G	In vivo	Soft tissue sarcoma and osteosarcoma	mice	Immunodeficient	A nude mouse model of liver metastasis and in a subcutaneous human xenograft model of pancreatic cancer	Xenograft	[28]
2015	Imlygic	In vivo	Melanoma	mice	Immunodeficient	Nude—BALB/c—Subcutaneous injection of 2 × 10⁶ of the appropriate tumor cells and tumors allowed to develop to an average diameter of approximately 0.5 cm	Xenograft	[29, 30]
2015	Imlygic	In vivo	Melanoma	mice	N/A	C57BL/6—Harding–Passey melanoma cells (1 × 10⁶) were injected subcutaneously in shaved areas of the flank, bilaterally	Xenograft	[29, 30]
2017	Kymriah	Ex-vivo	Relapsed B-cell acute lymphoblastic leukemia	mice	Immunodeficient	An immunodeficient NOD/Shi-scid IL-2Rγ null human leukemia xenograft mouse model—no lymphoma animal model was developed and tested as a proof of concept	Xenograft	[31, 32]
2017	Yescarta	Ex-vivo	Relapsed or Refractory large B-cell lymphoma	Without animal model	–	No lymphoma animal model was developed and tested as a proof of concept	–	[33]
2020	Tecartus	Ex-vivo	Relapsed/refractory mantle cell lymphoma	Without animal model	–	There are no representative in vitro assays, ex vivo models, or in vivo models	–	[34]
2021	Abecma	Ex-vivo	Multiple myeloma	mice	Immunodeficient	NSG mice with and without BCMA + xenografts	Xenograft	[35]
2021	ARI-0001	Ex-vivo	Adult relapsed/refractory acute lymphoblastic leukemia	mice	Immunodeficient	NOD/scid-IL-2Rnull—They were inoculated intravenously (i.v.) with 0.3 × 10⁶ GFP-NLuc Namalwa cells per mice	Xenograft	[36]
2021	Breyanzi	Ex-vivo	Relapsed or refractory diffuse large B-cell lymphoma; follicular lymphoma	mice	Immunodeficient	Raji xenograft athymic nude—No lymphoma animal model was developed and tested as a proof of concept. More information is given in the source	xenograft	[37, 38]
2021	Carteyva	Ex-vivo	Relapsed or refractory diffuse large B-cell lymphoma	N/A	N/A	No more information was found for this product	N/A	N/A
2021	Delytact	In vivo	Malignant Glioma	mice	Immunocompetent & athymic	Dissociated 005 GSCs (2–5 × 10⁴ cells) in 3 μL PBS were implanted stereotaxically into the striatum to generate orthotopic intracranial tumors	Xenograft	[39, 40]
2022	Adstiladrin	In vivo	Bladder cancer	mice	Immunodeficient	An orthotopic mouse model of human bladder cancer	Xenograft	[41]
2022	Adstiladrin	In vivo	Bladder cancer	monkey	N/A	No more information was found for this model	N/A	[41]
2022	Carvykti	Ex-vivo	Relapsed or refractory multiple myeloma	mice	Immunodeficient	NOG mice aged 6–8 weeks were injected s.c. with NCI-H929 cells (5 × 10⁶ cells) and tumor volume measured under blind conditions twice/week by caliper	Xenograft	[42]
2022	Carvykti	Ex-vivo	Relapsed or refractory multiple myeloma	monkey	N/A	i.v. injection of BCMA-TCB2	Xenograft	[42]

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ISSN: 2233-7660

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