RESOURCES

RESEARCH AREA: Autoimmune Cardiovascular Disease Cell and Gene Therapy Infectious Disease Metabolism Neurology Oncology Other Rare Disease Respiratory Disease

November 10, 2025

MHC Knock-in Mouse Models for Preclinical Evaluation of Cancer mRNA Vaccines

mRNA cancer vaccines represent a next-generation immunotherapy platform with the potential to elicit robust, antigen specific immune responses. Upon in vivo delivery, mRNA vaccines are taken up by antigen-presenting cells (APCs), translated into the tumor associated antigen and presented via MHC molecules to activate adaptive immunity. However, standard mouse models with murine MHC molecules are not suitable for evaluating these MHC-restricted immune responses. GemPharmatech has developed a series of MHC knock-in (KI) models across multiple mouse backgrounds including immunocompetent mice B6, BALB/c, CB6F1 and immunodeficient NCG derived mice. These models express human HLA molecules, enabling the accurate evaluation of antigen presentation, immunogenicity, and therapeutic efficacy of mRNA vaccines. Our diverse portfolio of MHC knock-in mouse models provide more versatile and translationally relevant platforms for preclinical testing of mRNA-based cancer vaccines and combination immunotherapies. These models bridge the gap between murine and human immune systems, enhancing predictive power in vaccine development pipelines.
November 10, 2025

NCG-hIL15 and NCG-hIL2 Mice - Excellent Models for Human Immune Reconstitution of NK Cells

Natural Killer (NK) cells play a critical role in antitumor immunity through direct cytotoxic activity and secretion of immunoregulatory cytokines. They are particularly important in Antibody-Dependent Cell-Mediated Cytotoxicity (ADCC), a key mechanism often leveraged by therapeutic antibodies. However, the conventional immunodeficient mouse models show limited capacity to support NK cell engraftment, restricting their translational relevance. To overcome this limitation, we developed two humanized cytokine-expressing immunodeficient NCG mouse models: NCG-hIL2 and NCG-hIL15, engineered to express human IL-2 and IL-15, respectively. NCG-hIL2 was well suited for evaluating ADCC antibodies like Trastuzumab, Margetuximab, Rituximab and Blinatumomab. Both NCG-hIL2 and NCG-hIL15 model offer powerful tools for investigating NK cell biology, cytokine-driven development, and immunotherapy evaluation.
October 30, 2025

Application of Psoriatic Arthritis Mouse Models in Preclinical Pharmacodynamic Evaluation

Psoriatic arthritis (PsA) is a complex inflammatory disorder that affects up to 30% of psoriasis patients. Its heterogeneous clinical manifestations and lack of standardized diagnostic criteria pose significant challenges for therapy development. Although targeted therapies have advanced in recent years, the unclear pathogenesis and patient variability highlight the need for robust preclinical models. To address this, we established two inducible PsA mouse models designed to support drug efficacy testing and accelerate therapeutic innovation.
October 30, 2025

Phenotypic and Transcriptomic Divergence in C3 Glomerular Mouse Models: Insights into Immune and Clinical Sex Correlates

Complement component C3 is central to the alternative pathway (AP) and plays a key role in the pathogenesis of C3 glomerulopathy (C3G), a rare renal disease characterized by complement dysregulation. To model human-specific complement activity, we developed two humanized C3 mouse models: B6-hC3 (expressing a human C3 transgene) and B6-hC3 mC3 KO (expressing a human C3 transgene with knockout of mouse C3). These models were designed to promote unregulated C3 activation of the alternative pathway (AP) and reproduce C3G-like phenotypes. Through longitudinal phenotyping and RNA sequencing, we sought to elucidate molecular mechanisms underlying disease development and investigate sex-associated gene expression signatures.
October 30, 2025

A Novel Preclinical Tool for Evaluating CD20 Antibody Efficacy Based on BAFF/CD20 Dual-Target Humanized Mice

CD20, a well-characterized B-cell surface marker, is central to B-cell activation and differentiation. In addition, it contributes to the pathogenesis of B-cell-mediated diseases, including lymphomas, rheumatoid arthritis (RA), and systemic lupus erythematosus (SLE). Due to its selective expression on B cells, CD20 is an attractive target for therapeutic antibodies. Previously, we established an SLE model in transgenic mouse strains overexpressing human BAFF on a C57BL/6 mouse background (B6-hBAFF, T036794). To extend this platform for CD20-targeted drug evaluation, we generated a dual-humanized model by crossing B6-hBAFF with CD20 humanized mice (BALB/c-hCD20, T057498), resulting in the BAFF/CD20 dual-humanized mice (T065937).
June 23, 2025

Semaglutide and Lisinopril Exerts Therapeutic Benefits in a Genetically Engineered Diabetic Nephropathy Mouse Model

Diabetic nephropathy (DN) is a major driver of chronic kidney disease, yet preclinical models often fail to replicate human disease severity. GemPharmatech addresses this by developing an improved translational DN mouse model that mimics progressive human kidney damage—enabling better evaluation of future therapies.
June 23, 2025

A Persistent Hypertension Model Developed on the AGT Humanized Mice

Hypertension, a major metabolic syndrome condition, has experienced a rapid increase in global prevalence. AGT (angiotensinogen), the key precursor in the RAAS pathway, serves as a prime target for RNAi therapies. GemPharmatech has developed an AGT humanized mouse model to advance hypertension research and developed a modified human RENIN virus to induce sustained high blood pressure in these mice.
June 18, 2025

Development of Preclinical Models for the Evaluation of Conventional and in vivo CAR-T Therapies

Traditional CAR-T therapies require expensive, complex ex vivo T-cell engineering. While in vivo CAR-T generation using lentiviral vectors offers a faster, cheaper alternative, preclinical testing is limited by graft-versus-host disease (GVHD) in immunodeficient mouse models. To overcome this, GemPharmatech developed the NCG-MHC-dKO mouse, featuring dual MHC I/II knockout to minimize GVHD while maintaining human immune cell engraftment. Testing an anti-CD19 CAR lentiviral vector in this model demonstrated successful in vivo CAR-T generation and potent tumor suppression, with no significant GVHD. The NCG-MHC-dKO model enables reliable, long-term evaluation of in vivo CAR-T therapies, enhancing translational research.
June 18, 2025

NCG-X: A Novel Mouse Model For Preclinical Studies Evaluating Humanized Erythroid Reconstitution

Hematopoietic stem cell (HSC) transplantation is critical for treating blood cancers and autoimmune diseases, but existing animal models rely on toxic preconditioning (irradiation/chemotherapy), leading to off-target effects that undermine research validity. To overcome this, we developed the NCG-X mouse by introducing a C-KIT (Val831Met) mutation into the NCG mouse. This model enables human HSC engraftment without irradiation or chemoablation, offering a promising platform for immune and erythroid reconstitution studies.

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