RESOURCES

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

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.
December 10, 2025

Next-Generation Mouse Models for T-cell Engager Preclinical Evaluation

T cell engagers (TCEs) act as a bridge that one end binding to CD3 on T cells and the other end binding to a tumor-associated antigen (TAA) on tumour cells. TCEs physically bring cytotoxic T cells directly next to the tumour cells. This forced proximity triggers the T cells to activate, proliferate and destroy the cancer cells. TCEs redirect the immune responses to eliminate the tumour, independent of TAA presentation. The NCG-MHC-dKO mouse is developed by knocking out both murine MHC class I and class II molecules in NCG mice. GvHD in NCG-MHC-dKO mice is attenuated and the survival of huPBMC-NCG-MHC-dKO mice is significantly prolonged. The growth of NCI-H69 human small cell lung cancer and MM.1S human multiple myeloma were significantly delayed by Tarlatamab in huPBMC-NCG mice. These findings indicate that human immune system reconstituted NCG mouse is an ideal model for TCE efficacy evaluation.
November 19, 2025

Development of Fully Human CD98hc Antibodies to Enhance Delivery of Therapeutic Drugs Across the Blood-Brain Barrier

CD98 heavy chain (CD98hc, also known as SLC3A2), is a type II transmembrane glycoprotein that serves as a critical component of heteromeric amino acid (AA) transporters through its interaction with different light-chain partners. CD98hc is highly expressed in the blood-brain barrier (BBB), where it facilitates receptor-mediated transcytosis and has emerged as a promising target for enhancing dug delivery across the BBB.
In this study, we screened a panel of fully human CD98hc antibodies generated from NeoMab™ transgenic mice. Lead candidates demonstrated robust endocytosis and transcytosis activity in vitro without altering CD98hc receptor abundance or amino acid transport function. These findings highlight fully human CD98hc antibodies as promising transcytosis-enabling modules (TEMs) for shuttling therapeutic agents across the BBB.

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