Customized Solutions for Gene-Edited Rabbit & Pig Model Development

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Driven by a commitment to bridging the gap between benchtop discovery and translational success, Protheragen’s platform provides end-to-end, customized gene-edited model development, specializing in both Gene-Edited Rabbit Models and Gene-Edited Pig Models, alongside comprehensive preclinical research services. Combining state-of-the-art precision genome editing with high-throughput characterization, our advanced translational capabilities empower global biopharmaceutical partners to accelerate candidate validation.

Overview of Gene-Edited Rabbit & Pig Models

Occupying crucial evolutionary and physiological niches between rodents and primates, genetically modified rabbit (medium-animal) and pig (large-animal) models successfully overcome the predictive limitations of traditional murine systems. By closely recapitulating human cardiovascular dynamics, metabolic pathways, renal architecture, ocular structures, and complex immune profiles, these human-relevant models enable high-fidelity modeling of sophisticated pathologies, ranging from advanced atherosclerosis and inherited cardiomyopathies to systemic metabolic syndromes and severe organ failure. Furthermore, their optimal physical scale uniquely supports longitudinal monitoring, repeated bio-sampling, surgical device testing, and xenotransplantation research.

Comparative Advantages of Rodent, Rabbit, and Pig Models

Applications of Gene-Edited Rabbit & Pig Models

Gene-edited rabbit and pig models serve as essential research engines across the drug discovery and development continuum, from target validation to preclinical efficacy and safety assessment. Their human-relevant physiology allows investigators to interrogate complex disease mechanisms and evaluate therapeutic modalities (including small molecules, biologics, and gene/cell therapies) with elevated translational confidence.

Cardiovascular Disease Research

Enabling the investigation of hypercholesterolemia, severe atherosclerosis, cardiac arrhythmias, and heart failure, providing a robust platform for evaluating lipid-lowering compounds, gene therapies, and anti-arrhythmia biologics in a translatable context.

Immunodeficiency, Xenotransplantation & Organ Engineering

Multi-gene knock-out and human-transgene knock-in pig and rabbit models serve as critical bio-platforms for studying cross-species organ transplantation, immune rejection mechanisms, hematopoietic reconstitution, and humanized tissue chimeras.

Neurological and Ophthalmic Disease Modeling

Providing superior systems for modeling neurodegenerative disorders and ocular pathologies, with larger brain and eye structures facilitating detailed anatomical, surgical, and functional assessments that are challenging in rodents.

Rare and Inherited Disease Research

Effectively recapitulating loss-of-function or gain-of-function phenotypes associated with rare genetic disorders (e.g., Duchenne muscular dystrophy, cystic fibrosis, tyrosinemia), providing a predictive platform to evaluate therapies.

Model Customization Capabilities Portfolio

Our platform offers custom gene-edited rabbit and pig model development services across a wide spectrum of disease indications and background strains (including New Zealand White Rabbit, Bama Miniature Pig, Tibetan Miniature Pig, etc.). Leveraging advanced genome-editing platforms, models can be engineered to carry precise genetic modifications, knockout, knock-in, point mutations, or conditional alleles, tailored to specific research objectives.

Integrated Preclinical Research Services

Beyond model development, our platform provides end-to-end preclinical research capabilities that leverage gene-edited rabbit and pig models for comprehensive therapeutic evaluation. These integrated services enable seamless progression from model generation through rigorous candidate assessment, delivering high-quality efficacy and safety data to guide translational strategy and support early-stage decision-making.

Case Study-DMD Knockout Rabbit Model

The dystrophin-deficient rabbit model (DMD-KO) was generated via targeted genome editing to disrupt the DMD gene, recapitulating the progressive skeletal and cardiac muscle degeneration characteristic of human Duchenne muscular dystrophy. This rabbit platform exhibited hallmark pathological features including elevated serum creatine kinase, diminished spontaneous ambulatory activity, and deteriorating cardiac systolic function, all of which progressed with age. Given the closer physiological and cardiorespiratory parallels to humans compared to murine systems, this model provided a highly translatable framework for evaluating both dystrophin-restoring therapeutics and adjunctive cardioprotective strategies.

Fig.1 Body weights of sex-matched wild-type (WT) and DMD-KO rabbits at 24 weeks of age. DMD-KO rabbits of both sexes showed significantly lower body weights than their WT counterparts at the same age. Data are presented as mean ± SEM (n=6; **p < 0.01).

At 24 weeks of age, the DMD-KO cohort consistently weighed less than WT littermates across both sexes, reflecting the generalized muscle wasting inherent to the dystrophic phenotype. Functional and biochemical evaluations further validated the model’s disease relevance. Spontaneous locomotor output, measured as walking steps recorded over a 1-hour interval, was markedly reduced in the KO animals relative to WT controls. Serum creatine kinase activity, a sensitive indicator of ongoing myofiber disruption, was substantially elevated in the KO group. Echocardiographic assessments carried out at 16 weeks of age revealed that left ventricular ejection fraction and fractional shortening were both significantly depressed in the DMD-KO rabbits.

Fig.2 Comprehensive phenotypic comparisons between DMD-KO and WT rabbits. (A) Spontaneous locomotor activity quantified by total walking steps during a 1-hour observation period. (B) Serum creatine kinase (CK) activity. (C) Left ventricular ejection fraction (EF) and (D) fractional shortening (FS). Data are presented as mean ± SEM (n=6; ***p < 0.001, **p < 0.01).

Case Study-FAH Knockout Pig Model

The FAH-deficient Tibetan miniature pig model (FAH^-/-) was generated via targeted genome editing to disrupt the FAH gene, establishing a human-relevant platform for Hereditary Tyrosinemia Type 1 (HT1). Managed with NTBC administration during gestation, FAH^-/- piglets subjected to NTBC withdrawal post-birth exhibited rapid physiological deterioration, severe hepatic necrosis, and complete loss of FAH protein expression, faithfully mimicking human liver failure and establishing an indispensable platform for liver humanization and gene therapy validation.

Fig.3 FAH protein expression profiling. Western blot and densitometric analyses confirm robust FAH protein expression in wild-type (WT) controls, whereas FAH expression is completely abolished in FAH^-/- pigs, confirming complete loss-of-function knockout. Data are presented as mean ± SEM (n=6, ***p < 0.001).

Why Choose Us?

• Precision Genome Editing Expertise: Builds on deep expertise in rabbit and pig reproductive biology and genome editing to reliably generate complex, disease-relevant genetic modifications across diverse background strains.
• End-to-End Service Integration: Integrates custom model creation, deep molecular phenotyping, colony expansion, and comprehensive preclinical testing into one unified platform to accelerate program timelines.
• Standardized Quality Assurance: Follows rigorous animal welfare protocols and standardized lab procedures to ensure reproducible, high-quality data across all studies.
• Customized Scientific Support: Provides tailored project management and study design, directly aligned with each client’s specific therapeutic indication and research goals.

Contact Us

Gene-edited rabbit and pig models represent a powerful and increasingly essential component of the translational research toolkit, offering physiological relevance and experimental versatility that bridge the gap between rodent models and human studies. With expertise in custom model development and integrated preclinical services, Protheragen is uniquely positioned to support therapeutic programs from target validation through preclinical studies. To discuss how gene-edited rabbit and pig models can accelerate your drug development program, please contact us.

All of our services and products are intended for preclinical research use only and cannot be used to diagnose, treat or manage patients.

• Rare Disease Diagnostics Development
• Rare Disease Therapeutics Development
• Drug Repurposing Service
• Rare Disease Model Development Services
• Drug Safety Evaluation Services
• Pharmacokinetics Services