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

May 18, 2026

Preclinical Evaluation Platform for mRNA Cancer Vaccines Based on Human MHC-Restricted Immune Response Assessment

mRNA-based cancer vaccines are an advanced immunotherapeutic strategy designed to generate potent, targeted immune activation against tumor antigens. However, standard mouse models with murine MHC molecules are not suitable for evaluating these MHC-restricted immune responses. To address this gap, GemPharmatech has implemented an integrated preclinical service platform for the evaluation of mRNA vaccine candidates, encompassing in vitro immunogenicity assessment, in vivo efficacy testing, and safety profiling. This platform employs a human immune cell-based co-culture system. For translational in vivo studies, GemPharmatech utilizes a panel of proprietary HLA-humanized mouse models. These human HLA molecules enable accurate evaluation of antigen presentation, immunogenicity, and therapeutic efficacy of mRNA vaccines. Therapeutic studies using TA-1 (mRNA vaccine) or anti-PD-1 in a patient-derived xenograft (PDX) model demonstrated that TA-1 or anti-PD-1 alone inhibited tumor growth, while combination therapy led to greater tumor inhibition.
May 18, 2026

Evaluating In Vivo CAR-T Efficacy in Humanized NCG-MHC-dKO Mouse Model with Delayed GVHD

PBMC-reconstituted mouse models are critical for evaluating in vivo CAR-T therapies, but their utility is limited by rapid-onset Graft-versus-Host Disease (GVHD). GemPharmatech employed a novel NCG-MHC-dKO model to mitigate GVHD, enabling prolonged assessment of anti-tumor efficacy for different in vivo CAR-T modalities. The NCG-MHC-dKO model was reconstituted with human PBMCs, which confirmed delayed GVHD onset. Mice bearing Nalm-6 tumors were treated with a single dose of either a lentiviral vector or an LNP formulation delivering a CD19-targeting CAR. Tumor growth was monitored via bioluminescence imaging. The PBMC-NCG-MHC-dKO model demonstrated a significantly extended observation window due to postponed GVHD. In this model, both lentiviral and LNP-based in vivo CAR-T therapies potently suppressed Nalm-6 tumor growth. Functional CAR-T cells generated in vivo were detected and found to expand in treated mice. The PBMC-reconstituted NCG-MHC-dKO model provides a robust and durable platform for in vivo CAR-T evaluation by alleviating early GVHD.
April 23, 2026

AACR 2026 Poster Resources

Our 16 AACR 2026 posters are now available for download!
April 20, 2026

Therapeutic Evaluation of a C3-Targeting Small Nucleic Acid Drug in a Spontaneous Humanized C3 Glomerulopathy Mouse Model

Complement component C3 is central to activation of the alternative complement pathway, and its dysregulation drives C3 glomerulopathy (C3G), a severe renal autoimmune disease lacking effective treatments. GemPharmatech established a humanized C3G mouse model (B6-hC3 mC3 KO) that overexpresses human C3 while lacking murine C3, spontaneously developing progressive glomerulopathy that recapitulates human C3G pathology. In this model, small nucleic acid drug treatment reduced serum C3 and C3a levels, suppressed alternative pathway hyperactivation, and decreased renal C3 deposition and inflammation, supporting its utility for mechanistic studies and preclinical evaluation of complement-targeted therapies.
April 20, 2026

Development and Therapeutic Evaluation of a Uniform Lymph Node Cell Transfer Model of Alopecia Areata

Alopecia areata (AA) is an autoimmune disorder characterized by non-scarring hair loss driven by cytotoxic T cell–mediated attack on hair follicles. GemPharmatech developed a lymph node cell (LNC) transfer–induced AA mouse model on the C3H background, in which recipients develop alopecia accompanied by increased infiltration of CD3+, CD8+, and NKG2D+ cells and upregulation of MHCI. This model recapitulates key features of autoimmune pathology and demonstrates responsiveness to Ritlecitinib and Tacrolimus, both of which promote hair regrowth and reduce inflammatory markers. Together, these findings establish a robust and reproducible preclinical platform for studying AA pathogenesis and evaluating therapeutic candidates.
April 20, 2026

A Novel Mouse Colitis Model Recapitulating Human T Cell and Myeloid Cell Reconstitution to Support Drug Evaluation in Inflammatory Bowel Disease

Inflammatory bowel disease (IBD) is an autoimmune disorder of the gastrointestinal tract with complex pathogenesis, and current animal models relying on endogenous immune activation have limited translational relevance for evaluating human biologics. GemPharmatech has established a humanized IBD model combining human immune system reconstitution (huHSC-NCG-M/hIL15) with dextran sulfate sodium (DSS) challenge to better reflect human disease. This model exhibits human T cell and myeloid cell reconstitution and DSS-induced colitis features, including weight loss, shortened colon length, reduced survival, and epithelial damage. It also responds to adalimumab treatment, supporting its utility for translational IBD research and therapeutic evaluation.
April 20, 2026

LPS-HDM-Induced Asthma Model: A Translational Platform for Neutrophilic Asthma and Glucocorticoid-Resistant Inflammation

Asthma is a heterogeneous inflammatory airway disease, and up to 50% of patients exhibit a non-Th2 phenotype characterized by neutrophilic inflammation and poor responsiveness to glucocorticoids. This glucocorticoid-resistant phenotype represents a major unmet clinical need, yet current preclinical models do not adequately capture its underlying mechanisms. To address this gap, GemPharmatech developed a murine model of neutrophilic asthma induced by combined exposure to lipopolysaccharide (LPS) and house dust mite (HDM). This model recapitulates key features of the phenotype, including airway neutrophilia, macrophage infiltration, and airway remodeling, and demonstrates resistance to dexamethasone treatment, reflecting glucocorticoid-refractory asthma. It provides a reproducible platform for studying neutrophilic asthma and supporting the development of precision therapies.
March 03, 2026

Construction And Pharmacological Evaluation Of A New Parkinson's Disease Mouse Model

Parkinson’s disease (PD) models typically capture only part of the disease spectrum: toxin-induced models produce dopaminergic neuron loss but lack α-synuclein pathology, while many transgenic models show aggregation without robust neurodegeneration or behavioral deficits. To address this limitation, we developed a humanized α-synuclein transgenic mouse expressing the familial PD mutations E46K and A53T on the C57BL/6J background under the neuron-specific PrP promoter.
This model demonstrates progressive α-synuclein aggregation in the substantia nigra from 1 month of age, accompanied by dopaminergic neuron loss, reduced dopamine levels, and reproducible motor deficits emerging at 2–3 months.
By integrating α-synuclein pathology, nigrostriatal degeneration, and motor impairment, this model provides a robust and translationally relevant platform for Parkinson’s disease research and therapeutic evaluation.
March 03, 2026

Unveiling FAD3T: A Groundbreaking Transgenic Mouse Model for Accelerating Alzheimer's Disease Research

Alzheimer’s disease (AD) research is often limited by long experimental timelines and models that fail to capture the full spectrum of human pathology. Here we present FAD3T, a next-generation transgenic mouse model on the C57BL/6J background carrying humanized APP, PSEN1, and MAPT genes with familial AD mutations.
FAD3T mice display translational plasma biomarkers as early as 1 month, including Aβ40, Aβ42, p-tau181, p-tau217, and neurofilament light chain (NfL). The model recapitulates a progressive amyloid cascade, followed by phosphorylated tau pathology and neuroinflammation by 6 months. These pathological changes translate into early spatial memory deficits, emerging at 3 months in females and 4 months in males, reflecting clinically relevant sex differences.
Overall, FAD3T integrates early biomarker translatability, progressive AD pathology, and sex-specific phenotypes, providing a powerful platform to accelerate Alzheimer’s disease research and therapeutic development.

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