Myelodysplastic Syndromes (MDS) and Related Models
Ace Therapeutics is a specialized preclinical contract research organization dedicated to supporting drug development for hematological disorders. Our in vivo model platform for myelodysplastic syndromes (MDS) and related diseases provides reliable tools for investigating disease mechanisms, evaluating candidate therapies, and generating translational data essential for early-stage drug development.
Myelodysplastic Syndromes (MDS) represent a group of heterogeneous clonal hematopoietic disorders characterized by ineffective hematopoiesis, peripheral cytopenias, and a variable risk of progression to acute myeloid leukemia (AML). With a median onset age of 70 years and over 50% of high-risk patients progressing to AML, MDS presents significant therapeutic challenges due to its complex pathophysiology and limited treatment options.
Current preclinical research faces three critical hurdles:
At Ace Therapeutics, we overcome these challenges through advanced genetically engineered models that capture the genetic diversity and clinical progression of human MDS.
We maintain the industry's most comprehensive collection of clinically relevant MDS models with extensive molecular and phenotypic validation:
| Model Designation | Background/Induction Method | Key Validation Parameters | Ideal For Evaluating |
| MDS-NSGS-SGM3 | Patient-derived xenograft in NSGS-SGM3 mice | - Human myeloid engraftment >80% - Cytogenetic stability monitoring - Progression to AML in high-risk subtypes |
- Targeted therapies - Antibody-based treatments - Stem cell modulators |
| GEMM-NUP98-HOXD13 | Conditional knock-in transgenic model | - Dysplastic morphology - Peripheral cytopenias - Altered myeloid:erythroid ratio |
- Epigenetic modulators - Differentiation-inducing agents |
| CEBPAmut Model | CRISPR-edited CEBPA mutations | - Neutrophil dysfunction (NE-WX/Y/Z biomarkers) - Characteristic blast morphology |
- Bcl-2 inhibitors - Combination therapies |
| TP53KO MDS/AML | TP53 knockout with del(7q) induction | - Therapy-related MDS progression - Chemoresistance profiling - IPSS-R correlation |
- Novel apoptosis inducers - Targeted protein degraders |
We conduct mechanism-focused efficacy studies using disease-relevant endpoints:
Our specialized PK/PD platforms address hematology-specific challenges:
Hematopoietic-specific safety assessment protocols:
Biomarker discovery and validation services:
All models are rigorously validated through phenotypic characterization and molecular profiling. Common validation endpoints include:
Where available, models are cross-referenced to clinical data from human MDS subtypes to ensure translational relevance.
Our MDS in vivo model services are particularly suitable for evaluating:
To learn more about our MDS in vivo model services or discuss a customized study plan, please contact our scientific team. We are committed to supporting your hematological drug development journey with scientifically validated, efficient, and customized preclinical solutions.
Can I request a customized study protocol using a specific MDS mutation model?
Yes. We offer flexible study designs and can incorporate models with specific genetic alterations, such as Tet2 or SRSF2 mutations, depending on your therapeutic target.
What types of endpoints can be included in the study?
Endpoints can include hematological parameters (e.g., hemoglobin, platelets), bone marrow cellularity, lineage-specific flow cytometry, cytokine levels, and molecular expression profiles.
Do you offer patient-derived xenograft (PDX) models for MDS?
Yes. We provide human MDS PDX services using immunodeficient mice. These are suitable for evaluating human-specific therapeutic candidates.
Can your models simulate the transformation from MDS to AML?
We offer models such as the MLL-AF9 model, which mimics leukemic transformation. These are useful for studying therapies aimed at halting disease progression.
What data deliverables will I receive?
You will receive a complete study report including raw data, statistical analysis, graphical summaries, and methodology documentation.
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Conclusion
Ace Therapeutics specializes in preclinical research for hematologic diseases, providing reliable models and cutting-edge analytical support to advance therapeutic discovery.
With years of scientific experience, we offer integrated solutions that help biopharma teams turn early-stage ideas into robust preclinical outcomes.