Rare Disease Models
- Background
- Models
- Features
- Application
- FAQ
Accelerating Rare Disease Breakthroughs with Precision Models
Enable your preclinical development of neurodegenerative, autoimmune, cardiovascular, renal and pediatric rare diseases with robust, well-characterized models for translation and gene therapy studies.
What Are Rare Diseases?
Rare diseases are defined as conditions with low prevalence in the population, often characterized by complex pathophysiology, limited therapeutic options, and high unmet medical need. Despite their individual rarity, over 7,000 rare diseases collectively affect hundreds of millions of patients worldwide.
Due to low commercial viability and lack of scientific understanding, relevant preclinical models for these diseases are limited. To enable the successful translation of therapies for rare diseases, it's vital to construct solid disease models that can be used for mechanism investigation, target validation, and therapy assessment.
Our Rare Disease Model Portfolio
Rare Neurodegenerative Diseases
Rare Neuromuscular & Autoimmune Diseases
- Experimental Autoimmune Myasthenia Gravis (EAMG) Model
- Scleroderma Model
- Duchenne Muscular Dystrophy (DMD) Model
Why Choose Us
Comprehensive Rare Disease Coverage
Broad portfolio spanning neurological, autoimmune, cardiovascular, renal, and ocular rare diseases.
Translational Research Focus
Models optimized for mechanism-of-action studies and clinical relevance.
Gene & Cell Therapy Ready
Compatible with AAV, RNA therapeutics, and immune-modulating strategies.
Advanced Immunology Platforms
Humanized and immune-competent systems for complex disease modeling.
Custom Study Support
Flexible study design tailored to your therapeutic development needs.
Application
Support in vivo evaluation of AAV- and LNP-based gene delivery for efficacy and safety assessment.
Gene Therapy
Enable testing of ASO, siRNA, and mRNA approaches for gene regulation in disease models.
RNA Therapeutics
Assess immune modulation, inflammatory responses, and biologics performance in vivo.
Immunotherapy
Identify and validate translational biomarkers linked to disease progression and treatment response.
Biomarker Discovery
Support target validation and mechanistic studies to strengthen translational confidence.
Mechanism of Action (MoA)
FAQ
What kinds of rare disease models are available?
Our rare disease model portfolio includes neurodegenerative, autoimmune, cardiovascular, renal, and ocular disease models suitable for translation studies.
Do your models support gene therapy and RNA studies?
Yes. Our models are compatible with both AAV and LNP mediated gene therapy as well as RNA therapeutics including ASOs, siRNA, and mRNA.
Can you customize study designs?
Absolutely. We offer flexible and customizable study designs that can be tailored to your specific research goals. Studies can be designed for target validation, efficacy studies, mechanism of action studies, and more.
What endpoints can be measured?
The endpoints available will depend on the disease model you choose. Potential endpoints include histopathology, biomarkers, immune profiling, PD readouts, and functional disease-related readouts.
Request a Study Design
Partner with us to design translational rare disease studies tailored to your therapeutic strategy and development stage. Our team provides expert guidance from model selection to in vivo study execution to support high-quality, decision-driving data.